Infantile-onset Spinal Muscular Atrophy Clinical Trial
Official title:
Expanded Access Program (EAP) to Provide Nusinersen to Patients With Infantile-onset Spinal Muscular Atrophy (SMA)
To provide access to nusinersen to eligible patients with Infantile-onset Spinal Muscular Atrophy (SMA) (consistent with Type 1) to address a high-unmet medical need.
The nusinersen expanded access program (EAP) is available at approved treatment centers in select territories. A doctor must decide whether nusinersen treatment is appropriate for each patient, based on the patient's medical history and program eligibility criteria. A full list of participating treatment centers is provided in the 'Contacts and Locations' section of this listing, and is regularly updated. Following local approval and official reimbursement of nusinersen in each territory, the EAP will close and patients will transfer to commercially available drug. ;