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"Duchenne%muscular%dystrophy"
- ... treat rare diseases like spinal muscular atrophy and Duchenne muscular dystrophy. ... other neurological disorders including other rare muscular disorders. Duchenne muscular dystrophy: Genetic therapies to improve muscle weakness Muscular dystrophies ...
- ... for three diseases in animal or cell models: Duchenne muscular dystrophy (in mice); sickle cell disease (in blood stem ...