Nusinersen
- Generic Name
- Nusinersen
- Brand Names
- Spinraza
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- 1258984-36-9
- Unique Ingredient Identifier
- 5Z9SP3X666
- Background
An antisense oligonucleotide that induces survival motor neuron (SMN) protein expression, it was approved by the U.S. FDA in December, 2016 as Spinraza for the treatment of children and adults with spinal muscular atrophy (SMA). It is adminstrated as direct intrathecal injection.
- Indication
Indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
- Associated Conditions
- Spinal Muscular Atrophy (SMA)
- Associated Therapies
- -
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