Targeting Neuromuscular Diseases with ASOs and siRNA Therapies
Neuromuscular diseases, such as Duchenne Muscular Dystrophy (DMD), Spinal Muscular Atrophy (SMA), and Amyotrophic Lateral Sclerosis (ALS), involve progressive muscle degeneration and neurological impairments, posing immense challenges for patients and caregivers. However, antisense oligonucleotides (ASOs) and small interfering RNA (siRNA) therapies offer promising solutions by targeting...
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