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Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls
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Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls
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Journal Article
Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls
2021
Overview
The pre-mRNA
SMN2
splicing modifier risdiplam was administered orally to 41 infants with type 1 spinal muscular atrophy. After 12 months of treatment, 12 infants were able to sit without support, and most had better scores on motor-performance scales than the upper limit of confidence intervals from historical controls.
Publisher
Massachusetts Medical Society
Subject
/ Age
/ Azo Compounds/adverse effects/therapeutic use
/ Babies
/ Dosage
/ Female
/ Genetics
/ Historically Controlled Study
/ Humans
/ Infant
/ Infants
/ Male
/ mRNA
/ Neuromuscular Agents/adverse effects/therapeutic use
/ Ostomy
/ Proteins
/ Pyrimidines/adverse effects/therapeutic use
/ Sciences de la santé humaine
/ Spinal Muscular Atrophies of Childhood/drug therapy/mortality/physiopathology
