About Infantile-onset X-linked spinal muscular atrophy
Infantile-onset X-linked spinal muscular atrophy is a rare disease catalogued by Orphanet (ORPHA:1145). It is associated with the UBA1 gene. Search below for active recruiting clinical trials from ClinicalTrials.gov, or visit the Orphanet expert page for specialist centres, natural history studies, and patient registries.
Clinical Trial Eligibility Tips
What to know before applying to Infantile-onset X-linked spinal muscular atrophy trials.
Search ClinicalTrials.gov for "Infantile-onset X-linked spinal muscular atrophy" or filter by Orphanet code ORPHA:1145 to find disease-specific studies.
Ask your specialist to refer you to a Centre of Expertise — Orphanet lists all accredited centres for this condition worldwide.
Natural history studies and patient registries often enrol before drug trials open — contact the Orphanet patient community first.
Patient Resources
Find recruiting Infantile-onset X-linked spinal muscular atrophy trials
Search 500,000+ studies from ClinicalTrials.gov, filtered for Infantile-onset X-linked spinal muscular atrophy. Updated daily.