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Nusinersen
- Nusinersen injection is used for the treatment of spinal muscular atrophy (an inherited condition that reduces muscle strength and movement) in infants, children, and adults. Nusinersen injection is in a class of medications called ...
- 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/Start Here ... Spinal Muscular Atrophy ... Spinal muscular atrophy (SMA) refers to a group of hereditary diseases that can damage and kill ...
- ... weakness progresses: Onasemnogene abeparvovec-xioi (Zolgensma) Risdiplam (Evrysdi) Nusinersen (Spinraza) These drugs work by increasing the amount ...
- Spinal Muscular Atrophy (SMA) (Muscular Dystrophy Association) - PDFSpinal Muscular Atrophy/Start Here ... Spinal Muscular Atrophy ... Muscular Dystrophy Association ... PDF
- Neuromuscular Disorders/Specifics ... Neuromuscular Disorders ... Degenerative Nerve Diseases/Start Here ... Degenerative Nerve Diseases ... National Institute of Neurological ...
- Spinal Muscular Atrophy (SMA) (Nemours Foundation)Spinal Muscular Atrophy/Children ... Spinal Muscular Atrophy ... Nemours Foundation ... Spinal Muscular Atrophy (SMA), SMA, scoliosis, curved spine, strophy, wasting, ...