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PX’Therapeutics
South Plainfield, New Jersey, United States
- Ownership
- Private
- Development stage
- Other
01
Overview
PTC Therapeutics has multiple approved products including Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy, Emflaza (deflazacort), commercialization rights for WAYLIVRA (volanesorsen) in Latin America, and collaborations on Evrysdi (risdiplam) for SMA. PTC518 showed mutant protein reduction in Phase 2 for Huntington’s disease.[1][2][3]
- Therapeutic areas
- NeurologyRare DiseasesMetabolic Disorders
- Modalities
- Small moleculeGene therapy
- Industry
- Biotech
02
Partnerships
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