Spinal muscular atrophy: who works on it

One card per institution. Filter by target, by country, or show only institutions in EU-13 (Horizon Europe widening) countries.

• All targets
• SMN1 4
• VEGF 0
• PARP 0
• TP53 0
• AR 0
• CFTR 1
• DMD 1
• G6PD 0
• HDAC 0
• HTT 1
• NF1 1
• NRF2 0

EU-13 is a filter, not a ranking: it only narrows the list.

24 of 200+ institutions, ordered by recent publications and open trials. This is not a ranking of quality.

Terminated, withdrawn and suspended trials with the reason the sponsor gave in the registry, quoted word for word.

• Withdrawn

  Home Monitoring of Adult Patients With SMA: a Pilot Multicenter Validation Study

  NCT05839145· Institut de Myologie, France· Updated 4 Nov 2026

  “funding issues”Registry text, quoted unchanged. Source

• Withdrawn

  Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).

  NCT07265232· Lantu Biopharma· Updated 25 Sept 2026

  “Study withdrawn prior to participant enrollment following a reassessment of the clinical development plan.”Registry text, quoted unchanged. Source

• Terminated

  Gut Peptides and Bone Remodeling in Children With Neuromuscular Disorders

  NCT07254988· University of Copenhagen· Updated 16 Apr 2026

  “Terminated since further recruitment proved difficult”Registry text, quoted unchanged. Source

• Withdrawn

  Chinese Regional Spinal Muscular Atrophy Patient Registration Study

  NCT05631418· The Children's Hospital of Zhejiang University School of Medicine· Updated 23 Feb 2023

  “Due to the lack of application materials, the normal research can not be carried out for the time being.”Registry text, quoted unchanged. Source

Trials whose registry record says individual participant data (IPD) will be shared.

• A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy

  NCT05115110· Hoffmann-La Roche· Source

• A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND)

  NCT04488133· Biogen· Source

• ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)

  NCT07448610· Novartis Pharmaceuticals· Source

• A Study of the Safety of Nusinersen (Spinraza®) and Its Effects on Motor Function in Koreans Living With SMA Treated During Their Usual Medical Care

  NCT04317794· Biogen· Source

• Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb

  NCT06300996· Marco Capogrosso· Source

• A Study to Learn About the Safety of BIIB115 Injections and How BIIB115 is Processed in the Bodies of Healthy Adult Male Volunteers and of Pediatric Participants With Spinal Muscular Atrophy Who Previously Took Onasemnogene Abeparvovec

  NCT05575011· Biogen· Source

• A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA)

  NCT07221669· Biogen· Source

• Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

  NCT05232929· Genentech, Inc.· Source

• A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec

  NCT07444450· Biogen· Source

• A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

  NCT03779334· Hoffmann-La Roche· Source

Find publications on this disease by a MeSH method term, for example a sequencing or imaging method.

• Organoids 1
• CRISPR-Cas Systems 4
• Gene Expression Profiling 1
• Whole Genome Sequencing 1
• Exome Sequencing 1
• Proteomics 4
• Metabolomics 1
• Mass Spectrometry 1
• Magnetic Resonance Imaging 13
• Machine Learning 1
• Cohort Studies 12

See which targets of this disease your abstract mentions, and go to the people working on them.