Status:
RECRUITING
UK SMA Patient Registry
Lead Sponsor:
Newcastle University
Collaborating Sponsors:
Biogen
Roche Pharma AG
Conditions:
Spinal Muscular Atrophy
SMA
Eligibility:
All Genders
Brief Summary
Spinal muscular atrophy (SMA) is a form of motor neuron disease, most commonly caused by a mutation in the survival motor neuron 1 gene (SMN1) which results in a wide disease spectrum affecting childr...
Detailed Description
Participants are asked to provide information by completing online questionnaires. The medical questionnaire asks specific questions about their SMA diagnosis and their condition, including their moto...
Eligibility Criteria
Inclusion
- All patients with a confirmed SMA diagnosis (or pending diagnosis) are eligible for inclusion. Diagnosis will be confirmed via genetic testing results
Exclusion
- There are no exclusion criteria for the registry
Key Trial Info
Start Date :
July 13 2008
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
May 31 2025
Estimated Enrollment :
800 Patients enrolled
Trial Details
Trial ID
NCT04292574
Start Date
July 13 2008
End Date
May 31 2025
Last Update
July 23 2024
Active Locations (1)
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1
John Walton Muscular Dystrophy Research Centre
Newcastle upon Tyne, United Kingdom, NE1 3BZ