Spinal Muscular Atrophy Clinical Trial
Official title:
A Managed Access Program (MAP) Cohort Treatment Protocol to Provide AVXS-101 to Patients With a Genetic Diagnosis of Spinal Muscular Atrophy (SMA) With 1, 2 or 3 Copies of SMN2
The purpose of this Cohort Treatment Protocol will allow access to AVXS-101 for eligible patients diagnosed with SMA.
The purpose of this Cohort Treatment Protocol will allow access to AVXS-101 for eligible
patients diagnosed with SMA.
The requesting Physician submits a request for access to drug (often referred to as
Compassionate Use) to AveXis which is reviewed and approved by the medical team experienced
with the drug and indication. The requesting Physician should refer to the latest
Investigator's Brochure (IB) or approved label for overview of drug including: nonclinical
and clinical experience, risk and benefits.
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|---|---|---|---|
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