Spinal muscular atrophy: who works on it

One card per institution. Pick a target or a country to narrow the list.

• 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

24 of 378 institutions, ordered by recent publications and open trials. This is not a ranking of quality.

Funded research projects that name this disease in their title, terms or summary and have not ended yet: who is funded, where, how much and until when. Organisations only; the funder's page has the details.

• NIH NINDS · U01

  Advancing Ex Vivo Gene Therapy: IND-Enabling Studies for ASAH1-related SMA-PME

  Cedars-Sinai Medical Center· Los Angeles, United States· US$825,159 awarded in fiscal year 2026· until Aug 2028

• NIH NIAMS · R01

  Osteotomy-Free Correction Of Acetabular Dysplasia: Characterization and Proper Patient Selection for the SHALE Procedure

  University of Arizona· Tucson, United States· US$715,778 awarded in fiscal year 2026· until Jul 2031

• NIH NINDS · R01

  Defining the role of GATA6 in SMA pathology

  Medical College of Wisconsin· Milwaukee, United States· US$588,303 awarded in fiscal year 2026· until Jul 2031

• NIH NIGMS · F32

  Development of a Chemotranscriptomic Profiling Technology to Deconvolute RNA On- and Off-targets

  University of Michigan at Ann Arbor· Ann Arbor, United States· US$76,300 awarded in fiscal year 2026· until Aug 2029

• NIH NINDS · R43

  Selection of a Lead Candidate for the Development of an ASO Therapeutic for NF1 Cognitive and Social Deficits

  Infixion Bioscience, Inc.· Escondido, United States· US$478,799 awarded in fiscal year 2026· until Aug 2028

• European Commission (Horizon Europe) · HORIZON-TMA-MSCA-PF-EF

  Multi-Material Additive Manufacturing of S690/Fe-Mn-Si-Cr-Ni SMA Joints with 30% Enhanced Static and Fatigue Life for Structural Applications through L-DED (MultiMAMSS)

  Politecnico di Milano· Milano, Italy· EU contribution €209,483· until Mar 2028

• European Commission (Horizon Europe) · HORIZON-TMA-MSCA-PF-GF

  Mechanisms of Skeletal Muscle Adaptation to Resistance Training in Spinal Muscular Atrophy (SMA-RT)

  Universita Degli Studi di Padova· Padova, Italy· EU contribution €396,991· until Jan 2029· 2 countries

• European Commission (Horizon Europe) · HORIZON-ERC-SYG

  Harnessing the splicing code for targeted control of gene expression (UNLEASH)

  Fundacio Centre de Regulacio Genomica· Barcelona, Spain· EU contribution €5,000,764· until May 2029· 3 countries

  The disease is named only in the funder's project summary, not in the title.

• European Commission (Horizon 2020) · ERC-COG

  Generation of Position Specific organoids to study human neuromuscular system development and disease (GPSorganoids)

  Max Delbrueck Centrum Fuer Molekulare Medizin in Der Helmholtz-Gemeinschaft (Mdc)· Berlin, Germany· EU contribution €2,799,375· until Dec 2027

  The disease is named only in the funder's project summary, not in the title.

9 of 90 running projects, newest first. Listed amounts together: US$49.0m + €8.4m.

Sources: NIH RePORTER (U.S. National Institutes of Health, public domain) and CORDIS (European Commission, CC BY 4.0). Changes: filtered by disease, organisations matched to ROR.

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 Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam

  NCT07444476· Biogen· Source

• A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies

  NCT05789758· Biogen· Source

• AAV9-ASAH1 intracisterna magna Gene Therapy (GNT0009) in Patients with Spinal Muscular Atrophy with Progressive Myoclonic Epilepsy (SMA - PME)

  2026-527323-10-00· Genethon· Source

• 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

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

• Late-Onset Tay-Sachs Disease With SMALED-Like Muscle MRI Pattern Despite a Distinct Clinical Phenotype.

  J Peripher Nerv Syst, Sept 2026

• Longitudinal spinal cord MRI in adult 5q-SMA: biomarker and pathophysiological insights.

  J Neurol, Jul 2026

• Muscle MRI as an Imaging Biomarker of Muscle Damage in Patients With Spinal and Bulbar Muscular Atrophy.

  Neurology, Jul 2026

• Electromyogram diagnosed chronic lumbar radiculopathy is highly associated with L4-S1 regional multifidus atrophy on magnetic resonance imaging.

  BMC Musculoskelet Disord, Jun 2026

• Tongue volume in spinal and bulbar muscular atrophy (SBMA): an AI-assisted automatic MRI analysis.

  J Neurol, Jun 2026

• Transforaminal Approach in SMA Patients: Cause of Nerve Injury or Treatment Option? A Case Report.

  Pain Med Case Rep, May 2026

• Hirayama disease: an uncommon cause of motor neuron disease.

  Arq Neuropsiquiatr, Mar 2026

• Quantitative Whole-Body Muscle MRI in Adults With Spinal Muscular Atrophy-A Sensitive Tool for Long-Time Evaluation of Disease Progression.

  Eur J Neurol, Mar 2026

• [Motor neuron diseases from a radiological perspective : Focus on amyotrophic lateral sclerosis].

  Radiologie (Heidelb), Feb 2026

• Longitudinal cohort study of muscle function and metabolic biomarkers in disease-modifying treatment of spinal muscular atrophy.

  J Neuromuscul Dis, Jan 2026

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