AANEM Meeting Highlights Advances in Gene Therapies for Muscular Dystrophies
The recent AANEM annual meeting in Savannah, Georgia, highlighted groundbreaking advancements in gene therapies for muscular dystrophies, focusing on patient selection and the challenges of pre-existing immunity to AAV vectors. Dr. Melissa Spencer emphasized the need for standardized assessment methods to identify suitable candidates for innovative treatments, particularly for Duchenne muscular dystrophy (DMD). With ongoing research and collaboration, the future of muscular dystrophy treatment looks promising, offering hope to affected individuals and their families.
Pfizer’s Gene Therapy Setback Raises Concerns
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Tragic Incident in Pfizer’s Duchenne Muscular Dystrophy Trial
A tragic incident occurred in a trial for Pfizer’s experimental gene therapy for Duchenne muscular dystrophy, where a young boy lost his life. Despite attributing the cause of death to cardiac arrest, Pfizer is still investigating the incident to determine the exact circumstances surrounding the tragedy.