Story perspectives
Breakthrough Therapies Advance for Myotonic Dystrophy, Huntington's, Parkinson's
9/27/2025
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Story summary
- Sanofi's SAR446268 gains fast track designation to treat juvenile and adult-onset myotonic dystrophy type 1.
- Sanofi's SAR402663 aims to treat neovascular age-related macular degeneration.
- uniQure's AMT-130 slowed Huntington's disease progression by 75% in a Phase 1/2 trial.
- Bayer's BlueRock Therapeutics began Phase III trial for bemdaneprocel for Parkinson's disease.
- eXmoor Pharma and Siam Bioscience formed a Southeast Asia collaboration.
