In Search of a Cure: The Development of Therapeutics to Alter the Progression of Spinal Muscular Atrophy

In Search of a Cure: The Development of Therapeutics to Alter the Progression of Spinal Muscular Atrophy

Until the recent development of disease-modifying therapeutics, spinal muscular atrophy (SMA) was considered a devastating neuromuscular disease with a poor prognosis for most affected individuals. Symptoms generally present during early childhood and manifest as muscle weakness and progressive para...

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Bibliographic Details
Main Authors: Kristine S. Ojala, Emily J. Reedich, Christine J. DiDonato, Stephen D. Meriney
Format: Article
Language:English
Published: MDPI AG 2021-02-01
Series:Brain Sciences
Subjects:
spinal muscular atrophy
motoneuron disease
neuromuscular disease
therapeutics
Online Access:https://www.mdpi.com/2076-3425/11/2/194

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