Risdiplam
- Generic Name
- Risdiplam
- Brand Names
- Evrysdi
- Drug Type
- Small Molecule
- Chemical Formula
- C22H23N7O
- CAS Number
- 1825352-65-5
- Unique Ingredient Identifier
- 76RS4S2ET1
- Background
Risdiplam is an orally bioavailable mRNA splicing modifier used for the treatment of spinal muscular atrophy (SMA). It increases systemic SMN protein concentrations by improving the efficiency of SMN2 gene transcription. This mechanism of action is similar to its predecessor nusinersen, the biggest difference being their route of administration: nusinersen r...
- Indication
Risdiplam is indicated for the treatment of spinal muscular atrophy (SMA).
- Associated Conditions
- Spinal Muscular Atrophy (SMA)
- Associated Therapies
- -
No news found
Drug Development Updates
Stay informed with timely notifications on clinical trials, regulatory changes, and research advancements related to this medication.
© Copyright 2024. All Rights Reserved by MedPath