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What is spinal muscular atrophy (SMA)?

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Spinal muscular atrophy most often affects babies and children and makes it hard for them to use their muscles. The nerve cells in the brain and spinal cord break down. The brain stops sending messages that control muscle movement. Your child may have trouble controlling head movement, sitting without help, and even walking. Over time, they also may have trouble swallowing and breathing.

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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What causes spinal muscular atrophy (SMA)?

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