New Frontiers in the Treatment of Spinal Muscular Atrophy
- PMID: 30376223
New Frontiers in the Treatment of Spinal Muscular Atrophy
Comment on
-
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.N Engl J Med. 2017 Nov 2;377(18):1713-1722. doi: 10.1056/NEJMoa1706198. N Engl J Med. 2017. PMID: 29091557 Clinical Trial.
-
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.N Engl J Med. 2017 Nov 2;377(18):1723-1732. doi: 10.1056/NEJMoa1702752. N Engl J Med. 2017. PMID: 29091570 Clinical Trial.
Similar articles
-
Current and future treatments of spinal muscular atrophy.Dev Med Child Neurol. 2020 Mar;62(3):267. doi: 10.1111/dmcn.14447. Epub 2019 Dec 26. Dev Med Child Neurol. 2020. PMID: 31879942 No abstract available.
-
Limited Assessment of Respiratory Muscle Response to Nusinersen Treatment in Infants with Spinal Muscular Atrophy.Am J Respir Crit Care Med. 2020 Mar 1;201(5):624. doi: 10.1164/rccm.201909-1854LE. Am J Respir Crit Care Med. 2020. PMID: 31747298 Free PMC article. No abstract available.
-
Reply to Chacko et al.: Limited Assessment of Respiratory Muscle Response to Nusinersen Treatment in Infants with Spinal Muscular Atrophy.Am J Respir Crit Care Med. 2020 Mar 1;201(5):624-626. doi: 10.1164/rccm.201910-2022LE. Am J Respir Crit Care Med. 2020. PMID: 31747301 Free PMC article. No abstract available.
-
Spinal muscular atrophies.Lancet. 1990 Aug 4;336(8710):280-1. Lancet. 1990. PMID: 1973974 Review. No abstract available.
-
Yeo and Darras: Extraneuronal Phenotypes of Spinal Muscular Atrophy.Ann Neurol. 2021 Jan;89(1):24-26. doi: 10.1002/ana.25930. Epub 2020 Oct 29. Ann Neurol. 2021. PMID: 33051879 Free PMC article. Review. No abstract available.
Publication types
MeSH terms
Substances
LinkOut - more resources
Medical