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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.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Nusinersen | Drug | Administered by intrathecal injection |
|
Key Inclusion Criteria:
Key Exclusion Criteria:
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Biogen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hospital Pablo Tobon Uribe | Medellín | 050036 | Colombia | |||
| Auckland City Hospital |
| Grafton |
| Auckland |
| 1023 |
| New Zealand |
| Auckland District Health Board ADHB | Grafton | Auckland | 1023 | New Zealand |
| Erciyes University Hospital | Kayseri | Anatolia | 38000 | Turkey (Türkiye) |
| Hacettepe University | Ankara | Central Anatolia | 06100 | Turkey (Türkiye) |
| Marmara Uni. Research & Educational Hospital | Kadıköy | Istanbul | 34722 | Turkey (Türkiye) |
| Medipol University Hospital | Istanbul | Marmara | 34214 | Turkey (Türkiye) |
| ID | Term |
|---|---|
| D009134 | Muscular Atrophy, Spinal |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
| D019636 | Neurodegenerative Diseases |
| D009468 | Neuromuscular Diseases |
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| ID | Term |
|---|---|
| C000590926 | nusinersen |
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