Selecting disease-modifying medications in 5q spinal muscular atrophy.

Cartwright, Michael S; Upadhya, Suraj

Cartwright, Michael S; Upadhya, Suraj.

Affiliation

Cartwright MS; Department of Neurology, Wake Forest School of Medicine, Winston-Salem, North Carolina, USA.
Upadhya S; Department of Neurology, Wake Forest School of Medicine, Winston-Salem, North Carolina, USA.

Muscle Nerve ; 64(4): 404-412, 2021 Oct.

Article in En | MEDLINE | ID: mdl-34231920

Subject(s)

Muscular Atrophy, Spinal/drug therapy; Neuromuscular Agents/administration & dosage; Oligonucleotides, Antisense/administration & dosage; Oligonucleotides/administration & dosage; Animals; Azo Compounds/administration & dosage; Azo Compounds/immunology; Biological Products/administration & dosage; Biological Products/immunology; Humans; Muscular Atrophy, Spinal/epidemiology; Muscular Atrophy, Spinal/immunology; Neuromuscular Agents/immunology; Oligonucleotides/immunology; Oligonucleotides, Antisense/immunology; Pyrimidines/administration & dosage; Pyrimidines/immunology; Recombinant Fusion Proteins/administration & dosage; Recombinant Fusion Proteins/immunology; Treatment Outcome

Key words

gene therapy; nusinersen; onasemnogene abeparvovec-xioi; risdiplam; spinal muscular atrophy

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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Oligonucleotides / Muscular Atrophy, Spinal / Oligonucleotides, Antisense / Neuromuscular Agents Limits: Animals / Humans Language: En Journal: Muscle Nerve Year: 2021 Document type: Article Affiliation country: United States Country of publication: United States

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