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How is spinal muscular atrophy (SMA) treated?

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The FDA has approved two medications to treat SMA: nusinersen (Spinraza) and onasemnogene abeparvovec-xioi (Zolgensma). Both are forms of gene therapy that affect the genes involved in SMA, and both have been shown to help infants reach motor milestones faster, like sitting up.

Other options for your child's treatment depends on their symptoms and the type of SMA they have. The goal is to help your child move better and be as independent as possible.

From: Spinal Muscular Atrophy WebMD Medical Reference

SOURCES:

Genetics Home Reference: "Spinal muscular atrophy."

KidsHealth: "Spinal Muscular Atrophy (SMA)."

MDA.org: "Spinal Muscular Atrophy."

Medscape: "Spinal Muscular Atrophy."

National Institute of Neurological Disorders and Stroke: "NINDS Spinal Muscular Atrophy Information."

Boston Children's Hospital: "Spinal Muscular Atrophy (SMA)."

Jennifer Trust for Spinal Muscular Therapy: "Adult Onset SMA."

Cure SMA: "Therapeutic Approaches."

New England Journal of Medicine : "Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy."

Muscular Dystrophy UK: "Developing a genetic therapy for spinal muscular atrophy."

Science Advances : "Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model."

Columbia University Department of Neurology: "Q&A on Spinraza Treatment for Spinal Muscular Atrophy (SMA) Patients."

Reviewed by Hansa D. Bhargava on May 23, 2019

SOURCES:

Genetics Home Reference: "Spinal muscular atrophy."

KidsHealth: "Spinal Muscular Atrophy (SMA)."

MDA.org: "Spinal Muscular Atrophy."

Medscape: "Spinal Muscular Atrophy."

National Institute of Neurological Disorders and Stroke: "NINDS Spinal Muscular Atrophy Information."

Boston Children's Hospital: "Spinal Muscular Atrophy (SMA)."

Jennifer Trust for Spinal Muscular Therapy: "Adult Onset SMA."

Cure SMA: "Therapeutic Approaches."

New England Journal of Medicine : "Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy."

Muscular Dystrophy UK: "Developing a genetic therapy for spinal muscular atrophy."

Science Advances : "Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model."

Columbia University Department of Neurology: "Q&A on Spinraza Treatment for Spinal Muscular Atrophy (SMA) Patients."

Reviewed by Hansa D. Bhargava on May 23, 2019

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