Sarepta’s ELEVIDYS: First Gene Therapy for Duchenne Muscular Dystrophy (DMD) Treatment
Duchenne muscular dystrophy (DMD) is a devastating genetic disorder that primarily affects boys, leading to progressive muscle degeneration and weakness. Historically, DMD has been managed through supportive care, steroids, and emerging pharmaceutical therapies, but there has been no cure. The...
What Does the Future Hold For Gene Therapy in the Duchenne Muscular Dystrophy (DMD) Treatment Market?
Duchenne muscular dystrophy (DMD) is a severe, X-linked genetic disorder characterized by progressive muscle degeneration and weakness. Affecting approximately 1 in 3,500 to 5,000 males globally, DMD is caused by mutations in the dystrophin gene, leading to the absence of dystrophin...
NASH: Meeting the Unmet Challenges in Liver Health
Meeting the Unmet: Nonalcoholic Steatohepatitis (NASH)
Nonalcoholic Steatohepatitis (NASH) is a progressive liver disease that represents a significant unmet medical need. Characterized by inflammation and damage in the liver, NASH is a severe form of nonalcoholic fatty liver disease (NAFLD), and its prevalence has been steadily increasing...
Nucleic Acids and Gene Therapies in Neuromuscular Disorders: Next-Generation Therapeutic Strategies
Neuromuscular disorders (NMDs) are a diverse group of conditions characterized by the progressive degeneration of muscle and nerve function. Among these, Duchenne muscular dystrophy (DMD) stands out as a devastating condition with no cure, resulting from mutations in the dystrophin gene....