Results 171 to 180 of about 1,729 (232)

The long-term efficacy of nusinersen on respiratory functions in children with symptomatic spinal muscular atrophy type 1. [PDF]

Turk J Med Sci
Kiliç MA, Kürekçi F, Kipoğlu O, Coşkun O, Karacebey BN, Katar S, Avci R, Genç HM, Çakir E, Yildiz EP.   +9 more
europepmc   +1 more source

Clinical and Genetic Profiles of 5q- and Non-5q-Spinal Muscular Atrophy Diseases in Pediatric Patients. [PDF]

Genes (Basel)
Nishio H, Niba ETE, Saito T, Okamoto K, Lee T, Takeshima Y, Awano H, Lai PS.   +7 more
europepmc   +1 more source

MCV/Q, Medical College of Virginia Quarterly, Vol. 13 No. 4 [PDF]

, 1977

core   +1 more source

Fatal outcomes following onasemnogene abeparvovec in advanced-stage spinal muscular atrophy. [PDF]

Gene Ther
Pongsakornkullachart P, Kulsirichawaroj P, Kongkasuwan R, Tovichien P, Jitwongwai S, Kanjanauthai S, Preeprem N, Limpaninlachat S, Sermpon N, Sanmaneechai O.   +9 more
europepmc   +1 more source

Families' Perceptions of Powered Mobility for Participation in Children With Spinal Muscular Atrophy Type 1: A Photovoice Study. [PDF]

Health Expect
Coello-Villalón M, Díaz-López CI, López-Muñoz P, Romay-Barrero H, Pacheco-da-Costa S, Plasencia-Robledo M, Longo E, Palomo-Carrión R.   +7 more
europepmc   +1 more source

Respiratory outcomes of onasemnogene abeparvovec treatment for spinal muscular atrophy: national real-world cohort study. [PDF]

Eur J Pediatr
Lavie M, Rochman M, Armoni Domany K, Golan Tripto I, Be'er M, Besor O, Sagi L, Aharoni S, Ginsberg M, Noyman I, Levine H.   +10 more
europepmc   +1 more source

Abnormal expression of myosin heavy chains in early postnatal stages of spinal muscular atrophy type I at single fibre level. [PDF]

Acta Myol
Hedberg-Oldfors C, Jennions E, Visuttijai K, Oldfors A.   +3 more
europepmc   +1 more source

Brazilian Portuguese version of the revised upper-limb module: cross-cultural adaptation and validation. [PDF]

Arq Neuropsiquiatr
Artilheiro MC, Iannicelli JR, Polido GJ, Fernandes TR, Almeida LA, Mendonça RH, Camelo CG, Moreno CAM, Zanoteli E.   +8 more
europepmc   +1 more source

Spinal muscular atrophy: Should we prescribe approved drugs to cohorts of patients in which they are unproven? [PDF]

Eur J Neurol
Fontanelli L, Di Rauso G, Bellini G.
europepmc   +1 more source

Successful pregnancy of an SMA type 3 sitter on Nusinersen therapy - a case report. [PDF]

BMC Neurol
Hiebeler M, Thiele S, Walter MC.
europepmc   +1 more source