Managed Healthcare Executive2h
Duchenne Muscular Dystrophy: The Brain Is Affected Too
Researchers, physicians, families and affected patients view Duchenne muscular dystrophy (DMD) as a neuromuscular condition. Children are diagnosed during the first years of life because they have ...
The American Journal of Managed Care7h
Survey Explores Needs of Patients With DMD, BMD Transitioning to Adulthood
Activities of daily life, education, and employment were areas of difficulty in the transition to adulthood for patients with ...
Children denied access to life-changing drug due to paperwork, say families
Givinostat should be available to people with Duchenne muscular dystrophy through a type of compassionate use scheme.
BioSpace7h
6 Rare Diseases That Have New FDA-Approved Treatments
ITF, IntraBio and Orchard are among the companies that have won FDA nods in the past year for Duchenne muscular dystrophy, ...
Managed Healthcare Executive21h
Solid Bioscience Reports Positive Early Data from Duchenne Gene Therapy Trial
Solid Biosciences reported recent positive data in developing their gene therapy candidate SGT-003. The phase 1/2 INSPIRE ...
Muscular Dystrophy News7h
What superheroes look like in life with Duchenne muscular dystrophy
Columnist Betty Vertin recognizes the admirable traits in those living with Duchenne muscular dystrophy — and their moms.
pharmaphorum22h
Sarepta bags key expansion to DMD gene therapy label in US
Sarepta’s gene therapy for Duchenne muscular dystrophy (DMD) will be an option for many more patients in the US after the FDA expanded the breadth of its approved labelling. Elevidys ...
Q1 Earnings Forecast for PepGen Issued By HC Wainwright
Equities research analysts at HC Wainwright issued their Q1 2025 EPS estimates for PepGen in a report issued on Monday, February 24th. HC Wainwright analyst A. Fein expects that the company will earn ...