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
- -
An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy
- First Posted Date
- 2012-10-11
- Last Posted Date
- 2021-04-13
- Lead Sponsor
- Biogen
- Target Recruit Count
- 34
- Registration Number
- NCT01703988
- Locations
- 🇺🇸
University of Utah School of Medicine, Salt Lake City, Utah, United States
🇺🇸Boston Children's Hospital, Boston, Massachusetts, United States
🇺🇸UT Southwestern Medical Center - Children's Medical Center Dallas, Dallas, Texas, United States
An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)
- First Posted Date
- 2011-12-19
- Last Posted Date
- 2021-02-18
- Lead Sponsor
- Biogen
- Target Recruit Count
- 28
- Registration Number
- NCT01494701
- Locations
- 🇺🇸
Children's Hospital Boston, Boston, Massachusetts, United States
🇺🇸Columbia University Medical Center, New York, New York, United States
🇺🇸UT Southwestern Medical Center - Children's Medical Center Dallas, Dallas, Texas, United States
Drug Development Updates
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