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- Most rare diseases are caused by a defect in a single gene. Gene therapies treat diseases by replacing or changing a gene that doesn’t work with one that ...
- Spinal Muscular Atrophy (National Institute of Neurological Disorders and Stroke)Spinal Muscular Atrophy/Start Here ... Spinal Muscular Atrophy ... Spinal muscular atrophy (SMA) refers to a group of hereditary diseases that can damage and kill ...
- Motor Neuron Diseases (National Institute of Neurological Disorders and Stroke)Neuromuscular Disorders/Specifics ... Neuromuscular Disorders ... Degenerative Nerve Diseases/Start Here ... Degenerative Nerve Diseases ... National Institute of Neurological ...