A Prospective Long-Term Registry of Patients with a Diagnosis of Spinal Muscular Atrophy (SMA).
M
Marcia Felker, MD
Primary Investigator
Enrolling By Invitation
100 years or below
All
Phase
N/A
500 participants needed
1 Location
Brief description of study
Primary objectives:
To assess the effectiveness of treatments for SMA
To assess long term safety
To assess overall survival of all patients with SMA
Secondary:
To assess healthcare utilization
To assess caregiver burden
To assess patient functional independence
Detailed description of study
Primary objectives:
To assess the effectiveness of treatments for SMA
To assess long term safety
To assess overall survival of all patients with SMA
Secondary:
To assess healthcare utilization
To assess caregiver burden
To assess patient functional independence
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Spinal Muscular Atrophy
-
Age: 100 years or below
-
Gender: All
Inclusion Criteria:
- Genetic confirmation of SMA
Exclusion Criteria:
- None
Updated on
01 Aug 2024.
Study ID: 1909090309, AVXS-101-RG-001
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