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- Understanding Corticosteroid Therapy in Duchenne Muscular Dystrophy (DMD) TreatmentCorticosteroid therapy is a cornerstone of Duchenne Muscular Dystrophy (DMD) treatment. These medications, such as prednisone and deflazacort, have shown significant benefits in slowing disease progression and prolonging ambulation in individuals with DMD. Corticosteroids work by reducing inflammation and suppressing muscle fiber damage, thus preserving muscle strength and function. However,...0 Commentarii 0 Distribuiri 1734 Views 0 previzualizare
- A New Hope for DMD: Sarepta’s ELEVIDYS Gene TherapySarepta’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...0 Commentarii 0 Distribuiri 494 Views 0 previzualizare
- Advances in Duchenne Muscular Dystrophy TreatmentCauses and Mechanism of Duchenne Muscular Dystrophy Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness. It is caused by the absence of dystrophin, a protein that helps keep muscle cells intact. The defective gene that causes DMD is located on the X chromosome, so it primarily affects boys. Girls can be carriers of the defective...0 Commentarii 0 Distribuiri 778 Views 0 previzualizare
- Breakthroughs in Alzheimer’s Disease Research: What’s on the Horizon?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...0 Commentarii 0 Distribuiri 483 Views 0 previzualizare
- NASH: Meeting the Unmet Challenges in Liver HealthNASH: 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...0 Commentarii 0 Distribuiri 433 Views 0 previzualizare
- Next-Gen Therapeutic Strategies: Nucleic Acids and Gene Therapies for Neuromuscular DisordersNucleic 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....0 Commentarii 0 Distribuiri 413 Views 0 previzualizare