Vyondys 53: What Does FDA Rejection Letter Mean?
The Food and Drug Administration published more than 200 letters it sent to companies after their medications were rejected on Thursday, drawing attention to...
Accredited Duchenne Centers (ADC) Aims to Provide Uniform and Up-to-date Care for All People Living with Duchenne Muscular Dystrophy
The World Duchenne Organization (WDO) launched the international Accredited Duchenne Centers (ADC) Program. The ADC Program's goal is to provide all individuals with Duchenne...
Wellcome Trust Donates £10 Million to Treat Rare Diseases
The new Synthetic Human Genome Project (SynHG) is receiving £10 million from Wellcome Trust to build the fundamental instruments, technologies, and techniques that will...
After Second Patient Death, Duchenne Muscular Dystrophy Communities Want Answers About Elevidys Gene Therapy
In a report released May 14 in Neurology, the AAN Guidelines Subcommittee cautions both proponents and detractors of the FDA-approved treatment for Duchenne muscular...
What’s Next for Patients with Duchenne Muscular Dystrophy (DMD) After Pharma Giants Pull Back from AAV-Based Gene Therapy Research?
Adeno-associated virus (AAV) vectors were once heralded as the answer to delivering gene therapies, but in recent years, their prominence has diminished. It was...