Infantile-onset Spinal Muscular Atrophy
Conditions
Keywords
Spinal Muscular Atrophy, SMA, SMN, SMNRx, ISIS-SMNRx, ISIS-SMN Rx, ISIS 396443, ISIS-396443, ENDEAR, IONIS-SMNRx, IONIS-SMN Rx, EMBRACE, NURTURE, IONIS, BIOGEN, SHINE, Nusinersen
Brief summary
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.
Detailed description
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.
Interventions
DRUGNusinersen
Administered by intrathecal injection
Sponsors
Biogen
Eligibility
Sex/Gender
ALL
Inclusion criteria
Key Inclusion Criteria: * Genetic documentation of 5q SMA homozygous gene deletion, homozygous mutation, or compound heterozygote. * Onset of clinical signs and symptoms at ≤ 6 months (180 days) of age, consistent with infantile onset, Type I SMA * Patient whose care in the opinion of the treating physician meets, and is expected to continue to meet, the guidelines set out in the 2007 Consensus Statement for Standard of Care in SMA Key
Exclusion criteria
* Patient is qualified to participate in an ongoing clinical trial with nusinersen * Participation in a prior nusinersen study * Previous exposure to nusinersen * History of brain or spinal cord disease that would interfere with the LP procedures or CSF circulation * Presence of implanted shunt for the drainage of CSF or implanted CNS catheter * Previous or current participation in a clinical trial with an investigational gene therapy for SMA * Participation in a study with an investigational therapy for SMA within 6 months or five half-lives of the investigational drug, whichever is the longer, prior to the first dose of nusinersen. NOTE: Other protocol defined Inclusion/
Countries
Colombia, New Zealand, Turkey (Türkiye)
Outcome results
None listed