113. 筋ジストロフィー Muscular dystrophy Clinical trials / Disease details
臨床試験数 : 646 / 薬物数 : 471 - (DrugBank : 105) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 170
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2015-002069-52-IE (EUCTR) | 11/12/2020 | 21/05/2019 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Casimersen Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Golodirsen Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | Serbia;United States;Taiwan;Greece;Spain;Ukraine;Ireland;Israel;Russian Federation;Italy;France;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Belgium;Poland;Bulgaria;Norway;Germany;Sweden | ||
| 2 | EUCTR2017-004625-32-BG (EUCTR) | 03/12/2020 | 28/10/2020 | A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: CASIMERSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: GOLODIRSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 150 | Phase 3 | United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Poland;Bulgaria;Germany;Sweden | ||
| 3 | EUCTR2017-004625-32-SE (EUCTR) | 17/06/2020 | 09/08/2018 | A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: CASIMERSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: GOLODIRSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 260 | Phase 3 | United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Poland;Bulgaria;Germany;Sweden | ||
| 4 | NCT04179409 (ClinicalTrials.gov) | February 18, 2020 | 12/8/2019 | A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of Casimersen, Eteplirsen and Golodirsen in Subjects With Duchenne Muscular Dystrophy Carrying Eligible DMD Duplications | A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of Casimersen, Eteplirsen and Golodirsen in Subjects With Duchenne Muscular Dystrophy Carrying Eligible DMD Duplications | Duchenne Muscular Dystrophy | Drug: Casimersen;Drug: Eteplirsen;Drug: Golodirsen | Kevin Flanigan | Sarepta Therapeutics, Inc. | Active, not recruiting | 6 Months | N/A | Male | 3 | Phase 2 | United States |
| 5 | EUCTR2015-002069-52-DK (EUCTR) | 26/11/2019 | 25/07/2019 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Casimersen Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Golodirsen Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | Serbia;United States;Taiwan;Greece;Spain;Ukraine;Ireland;Israel;Russian Federation;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Belgium;Poland;Bulgaria;Norway;Germany;Sweden | ||
| 6 | EUCTR2017-004625-32-FR (EUCTR) | 05/02/2019 | 23/07/2018 | A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: CASIMERSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: GOLODIRSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 260 | Phase 3 | United States;United Kingdom;Spain;Canada;Czech Republic;Sweden;Belgium;Ireland;Finland;Poland;Italy;Israel;France;Australia;Bulgaria;Germany | ||
| 7 | EUCTR2017-004625-32-BE (EUCTR) | 20/12/2018 | 10/09/2018 | A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: CASIMERSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: GOLODIRSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 150 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Finland;Spain;Ireland;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Sweden | ||
| 8 | EUCTR2017-004625-32-ES (EUCTR) | 07/09/2018 | 10/09/2018 | A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: CASIMERSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: GOLODIRSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 150 | Phase 3 | United States;Finland;Spain;Ireland;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Sweden | ||
| 9 | EUCTR2015-002069-52-BG (EUCTR) | 06/08/2018 | 11/06/2018 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Casimersen Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Golodirsen Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Israel;Russian Federation;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Bulgaria;Norway;Germany;Sweden | ||
| 10 | EUCTR2017-004625-32-GB (EUCTR) | 26/07/2018 | 23/01/2019 | A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: CASIMERSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: GOLODIRSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 260 | Phase 3 | United States;Finland;Spain;Ireland;Israel;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Poland;Australia;Germany;Sweden | ||
| 11 | EUCTR2015-002069-52-CZ (EUCTR) | 11/09/2017 | 14/03/2017 | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Casimersen Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Golodirsen Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Israel;Russian Federation;Italy;France;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Belgium;Poland;Bulgaria;Norway;Germany;Sweden | ||
| 12 | EUCTR2015-002069-52-BE (EUCTR) | 28/04/2017 | 05/09/2016 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Casimersen Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Golodirsen Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | United States;Serbia;Greece;Spain;Ukraine;Ireland;Russian Federation;Israel;Italy;France;Denmark;Australia;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Georgia;Norway;Germany;Sweden | ||
| 13 | EUCTR2015-002069-52-DE (EUCTR) | 07/02/2017 | 24/10/2016 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Casimersen Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Golodirsen Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Israel;Russian Federation;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Bulgaria;Norway;Germany;Sweden | ||
| 14 | NCT02500381 (ClinicalTrials.gov) | September 28, 2016 | 14/7/2015 | Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD) | A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: SRP-4045;Drug: SRP-4053;Drug: Placebo | Sarepta Therapeutics, Inc. | NULL | Active, not recruiting | 6 Years | 13 Years | Male | 229 | Phase 3 | United States;Argentina;Australia;Belgium;Bulgaria;Canada;Czechia;Denmark;France;Germany;Greece;Hungary;India;Ireland;Israel;Italy;Korea, Republic of;Mexico;Poland;Russian Federation;Serbia;Spain;Sweden;United Kingdom |
| 15 | EUCTR2017-004625-32-DE (EUCTR) | 26/07/2018 | A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: CASIMERSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: GOLODIRSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | NA | Female: no Male: yes | 260 | Phase 3 | United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Poland;Bulgaria;Germany;Sweden | |||
| 16 | EUCTR2017-004625-32-CZ (EUCTR) | 09/09/2019 | A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: CASIMERSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: GOLODIRSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 260 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Finland;Spain;Ireland;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Sweden | |||
| 17 | EUCTR2017-004625-32-PL (EUCTR) | 07/11/2019 | A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: CASIMERSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: GOLODIRSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | NA | Female: no Male: yes | 260 | Phase 3 | United States;United Kingdom;Spain;Canada;Czech Republic;Sweden;Belgium;Ireland;Finland;Poland;Italy;Israel;Australia;France;Bulgaria;Germany |