Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
What You Need to Know Before You Apply
What is the purpose of this trial?
Spinal muscular atrophy (SMA) is a neurogenetic disorder caused by a loss or mutation in the survival motor neuron 1 gene (SMN1) on chromosome 5q13, which leads to reduced SMN protein levels and a selective dysfunction of motor neurons. SMA is an autosomal recessive, early childhood disease with an incidence of 1:10,000 live births. SMA is the leading cause of infant mortality due to genetic diseases.The purpose of this registry is to assess the long term outcomes of patients with SMA in the context of advances in treatment options and also to characterize and assess long-term safety and effectiveness of OAV-101.
Who Is on the Research Team?
Novartis Pharmaceuticals
Principal Investigator
Novartis Pharmaceuticals
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Registry Enrollment
Participants are enrolled in a long-term observational registry to assess outcomes and safety of SMA treatments
Follow-up
Participants are monitored for safety and effectiveness after enrollment
What Are the Treatments Tested in This Trial?
Interventions
- Prospective observational registry
- Zolgensma
Find a Clinic Near You
Who Is Running the Clinical Trial?
Novartis Pharmaceuticals
Lead Sponsor
Dr. Vas Narasimhan
Novartis Pharmaceuticals
Chief Executive Officer since 2018
MD from Harvard Medical School
Dr. Shreeram Aradhye
Novartis Pharmaceuticals
Chief Medical Officer since 2021
MD
AveXis, Inc.
Lead Sponsor
United BioSource, LLC
Industry Sponsor