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.
The purpose of this Cohort Treatment Protocol will allow access to AVXS-101 for eligible patients diagnosed with SMA.