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pp-136

Capsules

Dossier type
CTD
Dossier status
Under development
Country of origin
United States
GMP approvals
FDA
Comments
Indication: Becker muscular dystrophy (ambulatory adults) Clinical stage: Phase 3 / Modality: Small molecule Mechanism / Target: Fast-skeletal and cardiac myosin modulator — reduces peak force during eccentric contractions, protecting fragile dystrophin-deficient muscle fibres from contraction-induced damage Route / form: Oral capsule (once daily) Differentiation: Addresses mechanical stress mechanism, not gene correction; applicable to all BMD patients regardless of mutation; potential to protect residual muscle function long-term
Manufacturer #39169

Clinical-stage muscle disease company developing precision myosin modulators. Targets skeletal and cardiac muscle diseases driven by abnormal sarcomere function. Wholly owned pipeline including Phase 3 asset for rare muscular dystrophy.
 

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