Disease Modifying Therapies for the Management of Children with Spinal Muscular Atrophy (5q SMA): An Update on the Emerging Evidence
Author:
Publisher
Informa UK Limited
Subject
Drug Discovery,Pharmaceutical Science,Pharmacology
Reference119 articles.
1. Natural history of denervation in SMA: Relation to age,SMN2 copy number, and function
2. Spinal Muscular Atrophies
3. Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for diagnosis, rehabilitation, orthopedic and nutritional care
4. Implementation of population-based newborn screening reveals low incidence of spinal muscular atrophy
5. Spinal muscular atrophy
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1. A Systematic Literature Review of the Natural History of Respiratory, Swallowing, Feeding, and Speech Functions in Spinal Muscular Atrophy (SMA);Journal of Neuromuscular Diseases;2024-09-03
2. Nutrition outcomes of disease modifying therapies in spinal muscular atrophy: A systematic review;Muscle & Nerve;2024-08-11
3. Nusinersen Improves Motor Function in Type 2 and 3 Spinal Muscular Atrophy Patients across Time;Biomedicines;2024-08-06
4. Healthcare resource utilisation and direct medical cost for individuals with 5q spinal muscular atrophy in Sweden;The European Journal of Health Economics;2024-04-20
5. No significant sex differences in incidence or phenotype for the SMNΔ7 mouse model of spinal muscular atrophy;Neuromuscular Disorders;2024-04
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