Dystrophy is any condition in which a part of the body weakens or wastes away. In muscular dystrophy, the weakness is in the muscles. An inherited genetic mistake prevents the body from making a ...
While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...
People with myotonic dystrophy type 1 (DM1), the most common adult-onset form of muscular dystrophy ... model that replicates ...
offering new pathways for understanding and treating Duchenne Muscular Dystrophy (DMD). A groundbreaking study has shed light on the complex interactions between dystrophin, a protein critical to ...
with an additional emphasis on rhabdomysarcomas (cancers with characteristics of skeletal muscle) and human muscular dystrophies. Other research areas include gene and cell therapies for muscular ...
When Raniya Scott was just two years old, her world changed with an unexpected diagnosis: Duchenne muscular dystrophy (DMD), ...
Pratteln, Switzerland, January 16, 2025 – Santhera Pharmaceuticals (SIX: SANN) announces that the National Institute for Health and Care Excellence (NICE) has issued positive Final Guidance that ...
Altruism values for treatments of rare, severe pediatric diseases have not been estimated. This study found the altruism value for a hypothetical new Duchenne muscular dystrophy treatment to be $80 ...
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