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Pharmacological Research|September 17, 2018
Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drugLaurie S Conklin, Jesse M Damsker, Eric P Hoffman, et al.
Brain : a Journal of Neurology|January 8, 2021
Natural history of Charcot-Marie-Tooth disease type 2A: a large international multicentre studyMenelaos Pipis, Shawna M E Feely, James M Polke, et al.
Muscle & Nerve|July 22, 2014
Ataluren treatment of patients with nonsense mutation dystrophinopathyKatharine Bushby, Richard Finkel, Brenda Wong, et al.
European Journal of Neurology|October 11, 2024
Long-term natural history in type II and III spinal muscular atrophy: a 4-year international study on the Hammersmith Functional Motor Scale ExpandedGiorgia Coratti, Francesca Bovis, Maria Carmela Pera, et al.
Neurology|February 13, 2020
A longitudinal study of CMT1A using Rasch analysis based CMT neuropathy and examination scoresVera Fridman, Stefan Sillau, Gyula Acsadi, et al.
American Journal of Respiratory and Critical Care Medicine|September 14, 2010
Strategic plan for lung vascular research: An NHLBI-ORDR Workshop ReportSerpil Erzurum, Sharon I Rounds, Troy Stevens, et al.
Neurology. Clinical Practice|June 27, 2024
Therapeutic Role of Nusinersen on Respiratory Progression in Pediatric Patients With Spinal Muscular Atrophy Type 2 and Nonambulant Type 3Federica Trucco, Deborah Ridout, Harriet Weststrate, et al.
Aging|March 31, 2010
Impact papers on aging in 2009Mikhail V Blagosklonny, Judy Campisi, David A Sinclair, et al.
JAMA Internal Medicine|October 20, 2020
Association Between Early Treatment With Tocilizumab and Mortality Among Critically Ill Patients With COVID-19Shruti Gupta, Wei Wang, Salim S Hayek, et al.
Annals of Neurology|September 14, 2020
Clinical Variability in Spinal Muscular Atrophy Type IIIGiorgia Coratti, Sonia Messina, Simona Lucibello, et al.
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