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.
Study Type
EXPANDED_ACCESS
Administered by intrathecal injection
Hospital Pablo Tobon Uribe
Medellín, Colombia
Auckland City Hospital
Grafton, Auckland, New Zealand
Auckland District Health Board ADHB
Grafton, Auckland, New Zealand
Erciyes University Hospital
Kayseri, Anatolia, Turkey (Türkiye)
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Hacettepe University
Ankara, Central Anatolia, Turkey (Türkiye)
Marmara Uni. Research & Educational Hospital
Kadıköy, Istanbul, Turkey (Türkiye)
Medipol University Hospital
Istanbul, Marmara, Turkey (Türkiye)