Medically reviewed by Smita Patel, DO Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed ...

BioMarin Pharmaceutical Inc. ( NASDAQ: BMRN) Q4 2024 Earnings Conference Call February 19, 2025 4:30 PM ET Traci McCarty - Head of IR Alexander Hardy - President and CEO Brian Mueller - EVP and CFO ...

Researchers have suggested that uniform evidence standards and reporting requirements could help speed patient access to cell and gene therapies and help ensure consistent safety and efficacy ...

People with Duchenne or Becker muscular dystrophy who had more siblings typically report being better prepared to transition to adult life, a study says.