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Combination therapy with onasemnogene and risdiplam in spinal muscular atrophy type 1.
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Combination therapy with onasemnogene and risdiplam in spinal muscular atrophy type 1.
Oechsel KF, Cartwright MS. Combination therapy with onasemnogene and risdiplam in spinal muscular atrophy type 1. Muscle Nerve. 2021 Oct; 64(4):487-490.
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PubMed
subject areas
Azo Compounds
Biological Products
Combined Modality Therapy
Drug Therapy, Combination
Female
Humans
Infant
Male
Pyrimidines
Recombinant Fusion Proteins
Retrospective Studies
Spinal Muscular Atrophies of Childhood
authors with profiles
Michael S. Cartwright MD