DDrare: Database of Drug Development for Rare Diseases

3. 脊髄性筋萎縮症
［臨床試験数：179，薬物数：102（DrugBank：26），標的遺伝子数：52，標的パスウェイ数：78］

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.

Drug = 2011-2012 seasonal flu vaccine Intramuscular; 2011-2012 seasonal flu vaccine Subcutaneous; 4-Aminopyridine; 4-aminopyridine; 4-cholesten-3-one, oxime; 4-cholesten-3-one,oxime; AVXS-101; AVXS-101 (previously known as scAAV9.CB.SMN); Adipose derived mesenchymal stem cell; Air; Amifampridine; Amifampridine Phosphate; Amifampridine Phosphate 10 MG Oral Tablet; Biocarn; Branaplam; Celecoxib; GH; HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY; Hydroxyurea; ISIS 396443; ISIS 396443 (BIIB058); ISIS 396443, BIIB058; Itraconazole; LEVOCARNITINE; LMI070; Leuprolide; Levocarnitine; Mestinon; Midazolam; N.a.; NUSINERSEN; NUSINERSEN SODIUM; Nerve Growth Factor; Norditropin SimpleXx; Norditropin SimpleXx 15 mg/1.5 ml; Nursinersen; Nusinersen; Nusinersen Injectable Product; Nusinersen Treatments; OLESOXIME; ONASEMNOGENE ABEPARVOVEC; Olesoxime; Onasemnogene Abeparvovec-xioi; Onasemnogene abeparvovec; Onasemnogene abeparvovec-xioi; Orfiril Saft; Other: Monthly assessments of functional motor abilities by a trained therapist; Other: Prospective observational registry; PARACETAMOL; Paracetamol; Paracetamol 120Mg/5mL Oral Suspension; Phenylbutyrate; Phosphate; Placebo; Procedure: Air stacking manuever; Procedure: Sham Procedure; Procedure: Sham procedure; Pyridostigmine; Pyridostigmine Bromide; Pyridostigmine bromide; RO6885247; RO6885247/F02; RO6885247/F03; RO7034067; RO7034067 / F12; RO7034067 / F13; RO7034067/F06 + solvente (RO; RO7034067/F06 with solvent (RO7034067/F08); RO7034067/F07 + solvente (RO; RO7034067/F07 with solvent (RO7034067/F09); RO7034067/F12; RO7034067/F13; RO7090919; RVG 03820; Reldesemtiv; Reldesemtiv 150 mg; Reldesemtiv 450 mg; Riluzole; Risdiplam; SESAME OIL, REFINED; SMART; SRK-015; Salbutamol; Sesame oil; Sodium phenylbutyrate; Sodium phenylbutyrate (NaPB); Somatotropin; Somatropin; Spinraza; Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide; TRO19622; Testosterone; VALPROATE SODIUM; VOLMAX*30CPR 4MG R.P.; VPA; Valproate; Valproate, Levocarnitine; Valproic Acid; Valproic Acid (VPA); Valproic Acid and Levocarnitine; Valproic acid; Zolgensma

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	NCT03648658 (ClinicalTrials.gov)	February 18, 2019	8/8/2018	Paracetamol Study in Patients With Low Muscle Mass	Pharmacokinetics and Safety of Treatment With Paracetamol in Children and Adults With Spinal Muscular Atrophy and Cerebral Palsy	SMA II;Cerebral Palsy	Drug: Paracetamol 120Mg/5mL Oral Suspension	Mette Cathrine Oerngreen	Elsass Foundation	Recruiting	6 Years	45 Years	All	48	Phase 4	Denmark

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

NCT03648658
(ClinicalTrials.gov)

February 18, 2019

8/8/2018

Paracetamol Study in Patients With Low Muscle Mass

Pharmacokinetics and Safety of Treatment With Paracetamol in Children and Adults With Spinal Muscular Atrophy and Cerebral Palsy

SMA II;Cerebral Palsy

Drug: Paracetamol 120Mg/5mL Oral Suspension

Mette Cathrine Oerngreen

Elsass Foundation

Recruiting

6 Years

45 Years

All

Phase 4

Denmark