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

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.

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

No trial in this view states that it shares participant data.

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

• Targeted antisense oligonucleotide treatment rescues developmental alterations in spinal muscular atrophy organoids.

  Nat Commun, Dec 2025

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