Pre-symptomatic spinal muscular atrophy: a proposed nosology
Author:
Affiliation:
1. Center for Experimental Neurotherapeutics, St. Jude Children’s Research Hospital , Memphis, TN 38105 , USA
2. Department of Neurology, University of Miami , Miami, FL 33136 , USA
Abstract
Publisher
Oxford University Press (OUP)
Subject
Neurology (clinical)
Link
https://academic.oup.com/brain/advance-article-pdf/doi/10.1093/brain/awac125/43968442/awac125.pdf
Reference11 articles.
1. Preventing amyotrophic lateral sclerosis: insights from pre-symptomatic neurodegenerative diseases;Benatar;Brain,2022
2. Spinal muscular atrophy—insights and challenges in the treatment era;Mercuri;Nat Rev Neurol,2020
3. Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study;De Vivo;Neuromuscul Disord,2019
4. Onasemnogene abeparvovec for presymptomatic infants with spinal muscular atrophy and two copies of SMN2: A phase III study;Strauss;Eur J Neurol,2021
5. RAINBOWFISH: A study of risdipalm in infants with presymptomatic spinal muscular atrophy (SMA);Servais;Neuromuscul Disord,2021
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1. Challenges and opportunities in spinal muscular atrophy therapeutics;The Lancet Neurology;2024-02
2. Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry;Journal of Neuromuscular Diseases;2024-01-18
3. Can the CHOP-INTEND be used as An Outcome Measure in the First Months of Age? Implications for Clinical Trials and Real World Data;Journal of Neuromuscular Diseases;2024-01-02
4. Daily Quantity and Kinematic Characteristics of Leg Movement in a Child With SMA (2 Copies SMN2);Pediatric Physical Therapy;2023-10
5. Nusinersen Treatment of Children with Later-Onset Spinal Muscular Atrophy and Scoliosis Is Associated with Improvements or Stabilization of Motor Function;Journal of Clinical Medicine;2023-07-26
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