Results 171 to 180 of about 19,843 (235)

An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials. [PDF]

CNS Drugs, 2019
Darras BT, Farrar MA, Mercuri E, Finkel RS, Foster R, Hughes SG, Bhan I, Farwell W, Gheuens S.   +8 more
europepmc   +1 more source

Genomic analysis of a spinal muscular atrophy (SMA) discordant family identifies a novel mutation in TLL2, an activator of growth differentiation factor 8 (myostatin): a case report. [PDF]

BMC Med Genet, 2019
Jiang J, Huang J, Gu J, Cai X, Zhao H, Lu H.   +5 more
europepmc   +1 more source

Systemic MV Gene Therapy Close to Clinical Trials for Several Neuromuscular Diseases [PDF]

Wells, D J
core   +1 more source

Multi-Study Proteomic and Bioinformatic Identification of Molecular Overlap between Amyotrophic Lateral Sclerosis (ALS) and Spinal Muscular Atrophy (SMA). [PDF]

Brain Sci, 2018
Šoltić D, Bowerman M, Stock J, Shorrock HK, Gillingwater TH, Fuller HR.   +5 more
europepmc   +1 more source

Spinal muscular atrophy (SMA) type II with scoliosis

, 2010
openaire   +1 more source

Eighteen Years with Spinal Muscular Atrophy (SMA) Type 1.

Tanaffos, 2014
Borja, Valencia, John Robert, Bach
openaire   +1 more source

Hip displacement management in spinal muscular atrophy in the era of disease modifying therapies: a Delphi consensus study in the UK. [PDF]

EClinicalMedicine
Vanegas MI, Baranello G, Norman-Taylor F, Kokkinakis M, UK SMA Hip Consensus Group.   +4 more
europepmc   +1 more source