Woman Ends Her Life After ALS Diagnosis, Chooses Medical Aid in Dying: 'I Don't Want More Time as a Patient'
Barbara Goodfriend, 83, opted for medical aid in dying after being diagnosed with ALS. The New Jersey widow ended her life in ...
BioSpace7d
Duchenne Muscular Dystrophy Space on Cusp of Pivotal Era
While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...
manilatimes5d
MDA Launches 75th Anniversary Campaign and Premieres New PSA to Shine a Light on Rare Diseases
MDA launches 75th Anniversary campaign and premieres new public service announcement (PSA) to mark 75 years of impact in the ...
Muscular Dystrophy Association Announces 2025 MDA National Ambassador Lily Sander
The Muscular Dystrophy Association (MDA) has selected Lily Sander from Charlotte, North Carolina, as its 2025 MDA National Ambassador. Sander joins MDA National Ambassador Ira Walker from Fort ...
Sarepta Reports Positive Phase 3 EMBARK Data For Elevidys In Duchenne Muscular Dystrophy
(RTTNews) - Sarepta Therapeutics, Inc. (SRPT) Monday reported positive topline results from Part 2 of the Phase 3 study, dubbed EMBARK, of its gene therapy Elevidys approved for the treatment of ...
The American Journal of Managed Care5d
EMBARK 2: Delandistrogene Moxeparvovec Shows Sustained Benefits in DMD
The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years ...
Healio11d
FDA grants fast-track designation to myotonic dystrophy type 1 treatment
The FDA has granted fast-track designation to an investigational antisense oligonucleotide to treat individuals with myotonic ...