| Phase 4 |
| NCT07448610 |
ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants… |
Novartis Pharmaceuticals |
Not Yet Recruiting |
| NCT05232929 |
Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) |
Genentech, Inc. |
Active, Not Recruiting |
| Phase 3 |
| NCT06971094 |
Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the… |
GeneCradle Inc |
Recruiting |
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Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal… |
Lantu Biopharma |
Recruiting |
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TMS for Exposure Therapy Resistant OCD |
Amsterdam UMC, location VUmc |
Recruiting |
| NCT07444476 |
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in… |
Biogen |
Recruiting |
| NCT04042025 |
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi |
Novartis Gene Therapies |
Active, Not Recruiting |
| NCT05626855 |
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous… |
Scholar Rock, Inc. |
Active, Not Recruiting |
| NCT05067790 |
A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as… |
Biogen |
Active, Not Recruiting |
| NCT05337553 |
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With… |
Biohaven Pharmaceuticals, Inc. |
Active, Not Recruiting |
| NCT05335876 |
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in… |
Novartis Pharmaceuticals |
Active, Not Recruiting |
| Phase 2/3 |
| NCT05115110 |
A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam… |
Hoffmann-La Roche |
Active, Not Recruiting |
| Phase 2 |
| NCT07047144 |
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and… |
Scholar Rock, Inc. |
Recruiting |
| NCT07287982 |
A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and… |
argenx |
Recruiting |
| Phase 1/2 |
| NCT05747261 |
Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN… |
Biocad |
Recruiting |
| NCT05824169 |
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal… |
GeneCradle Inc |
Recruiting |
| NCT06421831 |
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal… |
GeneCradle Inc |
Recruiting |
| NCT07070999 |
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular… |
Gemma Biotherapeutics |
Recruiting |
| NCT05614531 |
Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal… |
Guangzhou Jiayin Biotech Ltd |
Enrolling by Invitation |
| NCT05901987 |
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal… |
GeneCradle Inc |
Active, Not Recruiting |
| NCT07554924 |
A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal… |
Lanyue Biotech (Hangzhou) Co., Ltd. |
Not Yet Recruiting |
| NCT06288230 |
An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal… |
Lantu Biopharma |
Active, Not Recruiting |
| Early Phase 1 |
| NCT06888661 |
Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal… |
Guangzhou Jiayin Biotech Ltd |
Enrolling by Invitation |
| Not specified |
| NCT04010604 |
A Registered Cohort Study on SMA |
Wan-Jin Chen |
Recruiting |
| NCT05663008 |
Impairments of Neuro-muscular Communication in Motor-Neuron Disease: A Bio-Marker for… |
University of Dublin, Trinity College |
Recruiting |