3. Spinal muscular atrophy
179 clinical trials,   102 drugs   (DrugBank: 26 drugs),   52 drug target genes,   78 drug target pathways

Searched query = "Spinal muscular atrophy", "Myelopathic muscular atrophy", "SMA I", "Werdnig-Hoffman disease", "SMA II", "Dubowitz disease", "SMA III", "Kugelberg-Welander disease", "SMA IV"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
43 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2019-002663-10-GB
(EUCTR)
17/12/202001/09/2020Study of Nusinersen (BIIB058) in Participants With Spinal MuscularAtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen(BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
Product Name: Spinraza
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nursinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: Nursinersen
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
152Phase 2;Phase 3United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Ireland;Lebanon;Israel;Chile;Russian Federation;Colombia;Italy;Australia;Latvia;Korea, Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Argentina;Brazil;Poland;Germany
2EUCTR2019-002663-10-ES
(EUCTR)
06/05/202010/05/2020Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 2;Phase 3United States;Saudi Arabia;Estonia;Greece;Spain;Lebanon;Ireland;Russian Federation;Chile;Colombia;Italy;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Australia;Latvia;Korea, Republic of
3EUCTR2019-002663-10-HU
(EUCTR)
23/04/202005/03/2020Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 2;Phase 3United States;Saudi Arabia;Estonia;Spain;Ireland;Lebanon;Russian Federation;Chile;Colombia;Italy;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Australia;Latvia;Korea, Republic of
4EUCTR2019-002663-10-LV
(EUCTR)
27/03/202012/02/2020Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 2;Phase 3United States;Saudi Arabia;Estonia;Spain;Ireland;Lebanon;Russian Federation;Chile;Colombia;Italy;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Australia;Latvia;Korea, Republic of
5EUCTR2015-001870-16-GB
(EUCTR)
04/10/201629/10/2015A study for participants with Spinal Muscular Atrophy (SMA) who previously participated in nusinersen (ISIS 396443) investigational studiesAn Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: Nusinersen
Other descriptive name: ISIS 396443
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
292Phase 3United States;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2015-001870-16-BE
(EUCTR)
29/07/201623/05/2016A study for participants with Spinal Muscular Atrophy (SMA) who previously participated in nusinersen (ISIS 396443) investigational studiesAn Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443 (BIIB058)
INN or Proposed INN: Nusinersen
Other descriptive name: ISIS 396443
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
292Phase 3United States;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
7EUCTR2015-001870-16-SE
(EUCTR)
26/05/201622/01/2016A study for participants with Spinal Muscular Atrophy (SMA) who previously participated in nusinersen (ISIS 396443) investigational studiesAn Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: Nusinersen
Other descriptive name: ISIS 396443
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
292Phase 3United States;Hong Kong;Spain;Turkey;Italy;United Kingdom;France;Canada;Belgium;Australia;Germany;Japan;Sweden;Korea, Republic of
8EUCTR2015-001870-16-ES
(EUCTR)
08/03/201603/02/2016An extension study for patients with Spinal Muscular Atrophy who participated to the previous ISIS 396443 studiesAn Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443 Spinal Muscular Atrophy (SMA)
MedDRA version: 18.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
274United States;Taiwan;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
9EUCTR2015-001870-16-DE
(EUCTR)
08/03/201628/10/2015An extension study for patients with Spinal Muscular Atrophy who participated to the previous ISIS 396443 studiesAn Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443 Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: Nusinersen
Other descriptive name: ISIS 396443
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
292Phase 3United States;Hong Kong;Spain;Turkey;Italy;United Kingdom;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
10EUCTR2014-003657-33-GB
(EUCTR)
17/11/201519/06/2015A Study to assess the safety and tolerability of ISIS 396443 in participants with spinal muscular atrophy (SMA).A phase 2, randomized, double-blind, sham-procedure controlled study to assess the safety and tolerability and explore the efficacy of ISIS 396443 (BIIB058) administered intrathecally in subjects with spinal muscular atrophy who are not eligible to participate in the clinical studies ISIS 396443-CS3B or ISIS 396443-CS4 - EMBRACE Spinal Muscular Atrophy
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Biogen Idec Research LimitedNULLNot Recruiting Female: yes
Male: yes
16Phase 2United States;Germany;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT02594124
(ClinicalTrials.gov)
November 4, 201530/10/2015A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies.An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443Spinal Muscular AtrophyDrug: nusinersenBiogenNULLActive, not recruitingN/AN/AAll292Phase 3United States;Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;Taiwan
12EUCTR2013-004422-29-BE
(EUCTR)
22/10/201504/08/2015A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
111Phase 3United States;Taiwan;Hong Kong;Spain;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
13EUCTR2014-003657-33-DE
(EUCTR)
21/09/201505/05/2015A Study to assess the safety and tolerability of ISIS 396443 in participants with spinal muscular atrophy (SMA).A phase 2, randomized, double-blind, sham-procedure controlled study to assess the safety and tolerability and explore the efficacy of ISIS 396443 (BIIB058) administered intrathecally in subjects with spinal muscular atrophy who are not eligible to participate in the clinical studies ISIS 396443-CS3B or ISIS 396443-CS4 - EMBRACE Spinal Muscular Atrophy
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443 (BIIB058)
INN or Proposed INN: Nusinersen
Other descriptive name: ISIS 396443 (BIIB058)
Biogen Idec Research LimitedNULLNot RecruitingFemale: yes
Male: yes
16Phase 2United States;Germany;United Kingdom
14NCT02462759
(ClinicalTrials.gov)
August 19, 201514/5/2015A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).A Phase 2, Randomized, Double-blind, Sham-procedure Controlled Study to Assess the Safety and Tolerability and Explore the Efficacy of ISIS 396443 (BIIB058) Administered Intrathecally in Subjects With Spinal Muscular Atrophy Who Are Not Eligible to Participate in the Clinical Studies ISIS 396443-CS3B or ISIS 396443-CS4Spinal Muscular AtrophyDrug: Nusinersen;Procedure: Sham ProcedureBiogenNULLTerminatedN/AN/AAll21Phase 2United States;Germany
15EUCTR2014-002098-12-DE
(EUCTR)
05/08/201509/04/2015A Study of Multiple Doses of ISIS ISIS 396443 Delivered to Infants withGenetically Diagnosed and Presymptomatic Spinal Muscular AtrophyAn Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy - NURTURE Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443 (BIIB058)
INN or Proposed INN: Nusinersen
Other descriptive name: ISIS 396443 (BIIB058)
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
25Phase 2United States;Qatar;Taiwan;Canada;Turkey;Australia;Germany;United Kingdom;Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16EUCTR2014-001947-18-DE
(EUCTR)
07/07/201502/12/2014A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
117Phase 3France;United States;Hong Kong;Canada;Spain;Germany;Japan;Italy;United Kingdom;Korea, Republic of;Sweden
17EUCTR2014-002098-12-GB
(EUCTR)
10/06/201530/03/2015A Study of Multiple Doses of ISIS ISIS 396443 Delivered to Infants withGenetically Diagnosed and Presymptomatic Spinal Muscular AtrophyAn Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy - NURTURE Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
Product Name: Spinraza
Product Code: ISIS 396443 (BIIB058)
INN or Proposed INN: Nusinersen
Other descriptive name: ISIS 396443 (BIIB058)
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
25Phase 2United States;Qatar;Taiwan;Turkey;Australia;Germany;Italy;United Kingdom
18NCT02386553
(ClinicalTrials.gov)
May 20, 201527/2/2015A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular AtrophyAn Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy.Spinal Muscular AtrophyDrug: NusinersenBiogenNULLActive, not recruitingN/A6 WeeksAll25Phase 2United States;Australia;Canada;Germany;Italy;Qatar;Taiwan;Turkey;Argentina;Israel;United Kingdom
19EUCTR2014-002098-12-IT
(EUCTR)
12/05/201524/03/2015A Study of Multiple Doses of ISIS ISIS 396443 Delivered to Infants withGenetically Diagnosed and Presymptomatic Spinal Muscular AtrophyAn Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy - NURTURE Spinal Muscular Atrophy (SMA)
MedDRA version: 17.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443 (BIIB058)
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443 (BIIB058)
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
25United States;Qatar;Taiwan;Argentina;Turkey;Australia;Israel;Germany;United Kingdom;Italy
20EUCTR2014-001947-18-ES
(EUCTR)
08/04/201509/02/2015A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 18.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Isis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
117Phase 3France;United States;Hong Kong;Canada;Spain;Germany;Japan;Italy;United Kingdom;Korea, Republic of;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21EUCTR2014-001947-18-IT
(EUCTR)
12/03/201512/05/2015A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 18.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Isis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
117Phase 3France;United States;Hong Kong;Canada;Spain;Germany;Japan;United Kingdom;Italy;Korea, Republic of;Sweden
22EUCTR2014-001947-18-SE
(EUCTR)
03/02/201523/12/2014A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
117Phase 3France;United States;Hong Kong;Canada;Spain;Germany;Japan;Italy;United Kingdom;Korea, Republic of;Sweden
23EUCTR2013-004422-29-ES
(EUCTR)
20/01/201508/09/2014A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 17.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Isis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
111Phase 3Korea, Republic of;United States;Taiwan;Hong Kong;Spain;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Sweden
24EUCTR2013-004422-29-DE
(EUCTR)
08/01/201527/11/2014A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
111Phase 3United States;Taiwan;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
25EUCTR2013-004422-29-SE
(EUCTR)
09/12/201423/10/2014A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 18.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
111Phase 3United States;Taiwan;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26EUCTR2013-004422-29-GB
(EUCTR)
01/12/201429/07/2014A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy - Study to assess the Clinical Efficacy and Safety of ISIS 396443 Spinal Muscular Atrophy (SMA)
MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
111Phase 3United States;Taiwan;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
27NCT02292537
(ClinicalTrials.gov)
November 24, 201412/11/2014A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Later-onset Spinal Muscular AtrophySpinal Muscular AtrophyDrug: Nusinersen;Procedure: Sham procedureBiogenNULLCompleted2 Years12 YearsAll126Phase 3United States;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;United Kingdom
28EUCTR2013-004422-29-IT
(EUCTR)
13/11/201423/07/2014A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 17.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Isis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
111Phase 3United States;Taiwan;Hong Kong;Spain;Italy;United Kingdom;France;Canada;Belgium;Australia;Germany;Japan;Sweden;Korea, Republic of
29NCT02193074
(ClinicalTrials.gov)
August 19, 201414/7/2014A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular AtrophyA Phase 3, Randomized, Double-Blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Infantile-onset Spinal Muscular AtrophySpinal Muscular AtrophyDrug: nusinersen;Procedure: Sham procedureBiogenNULLTerminatedN/A210 DaysAll122Phase 3United States;Australia;Belgium;Canada;France;Germany;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;Hong Kong;Taiwan
30NCT02052791
(ClinicalTrials.gov)
January 201430/1/2014An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246)An Open-label Study to Assess the Safety and Tolerability of ISIS 396443 in Patients With Spinal Muscular Atrophy Who Previously Participated in 396443-CS2 or 396443-CS10Spinal Muscular AtrophyDrug: nusinersenBiogenNULLCompletedN/AN/AAll47Phase 1United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
31NCT01839656
(ClinicalTrials.gov)
May 8, 201322/4/2013A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)A Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Infantile-Onset Spinal Muscular AtrophySpinal Muscular AtrophyDrug: nusinersenBiogenNULLCompletedN/A210 DaysAll21Phase 2United States;Canada
32NCT01780246
(ClinicalTrials.gov)
January 201328/1/2013An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701)An Open-label Study to Assess the Safety and Tolerability of a Single Intrathecal Dose of ISIS 396443 in Patients With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1Spinal Muscular AtrophyDrug: nusinersenBiogenNULLCompleted2 Years15 YearsAll18Phase 1United States
33NCT01703988
(ClinicalTrials.gov)
October 20128/10/2012An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular AtrophyAn Open-Label, Dose Escalation Study to Assess the Safety, Tolerability and Dose-Range Finding of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Spinal Muscular AtrophySpinal Muscular AtrophyDrug: NusinersenBiogenNULLCompleted2 Years15 YearsAll34Phase 1;Phase 2United States
34NCT01494701
(ClinicalTrials.gov)
November 30, 201113/12/2011An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)An Open-label, Escalating Dose Study to Assess the Safety, Tolerability and Dose-range Finding of a Single Intrathecal Dose of ISIS 396443 in Patients With Spinal Muscular AtrophySpinal Muscular AtrophyDrug: nusinersenBiogenNULLCompleted2 Years14 YearsAll28Phase 1United States
35EUCTR2017-000327-27-Outside-EU/EEA
(EUCTR)
26/01/2017An Open-Label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular AtrophyAn Open-Label, Dose Escalation Study to Assess the Safety, Tolerability and Dose-Range Finding of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA);Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: nusinersen
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNAFemale: yes
Male: yes
34Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
36EUCTR2015-001870-16-FR
(EUCTR)
03/05/2016An extension study for patients with Spinal Muscular Atrophy who participated to the previous ISIS 396443 studiesAn Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443. Spinal Muscular Atrophy (SMA)
MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNAFemale: yes
Male: yes
274Phase 3United States;Taiwan;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
37EUCTR2014-001947-18-GB
(EUCTR)
19/12/2014A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 17.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Isis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
117Phase 3France;United States;Hong Kong;Canada;Spain;Germany;Japan;Italy;United Kingdom;Korea, Republic of;Sweden
38EUCTR2017-000621-12-Outside-EU/EEA
(EUCTR)
15/02/2017A Study to Assess the Efficacy, Safety and Pharmacokinetics of IONIS SMNRx in Infants With Spinal Muscular AtrophyA Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Infantile-Onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA);Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: NUSINERSEN
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNAFemale: yes
Male: yes
20Phase 2United States;Canada
39EUCTR2019-002663-10-DE
(EUCTR)
23/10/2020Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLNAFemale: yes
Male: yes
152Phase 2;Phase 3United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Ireland;Lebanon;Israel;Chile;Russian Federation;Colombia;Italy;France;Australia;Latvia;Korea, Republic of;Turkey;Hungary;Mexico;Canada;Argentina;Brazil;Poland;Germany
40EUCTR2019-002663-10-GR
(EUCTR)
02/11/2020Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLNAFemale: yes
Male: yes
152Phase 2;Phase 3Canada;Argentina;Brazil;Poland;Germany;United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Ireland;Lebanon;Israel;Chile;Russian Federation;Colombia;Italy;France;Australia;Latvia;Korea, Republic of;Turkey;Hungary;Mexico
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
41EUCTR2019-002663-10-IE
(EUCTR)
04/03/2020Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLNAFemale: yes
Male: yes
152Phase 2;Phase 3United States;Taiwan;Saudi Arabia;Estonia;Spain;Lebanon;Ireland;Turkey;Russian Federation;Chile;Israel;Colombia;Italy;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Australia;Latvia;Korea, Republic of
42EUCTR2014-001947-18-FR
(EUCTR)
18/06/2015A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 18.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Isis Pharmaceuticals, Inc.NULLNAFemale: yes
Male: yes
117Phase 3United States;France;Hong Kong;Canada;Spain;Germany;Japan;Italy;United Kingdom;Korea, Republic of;Sweden
43EUCTR2019-002663-10-PL
(EUCTR)
11/03/2020Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLNAFemale: yes
Male: yes
152Phase 2;Phase 3United States;Taiwan;Saudi Arabia;Estonia;Spain;Lebanon;Ireland;Turkey;Russian Federation;Chile;Israel;Colombia;Italy;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Australia;Latvia;Korea, Republic of