Results 131 to 140 of about 6,197 (153)

Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial

Nature Medicine, 2022
Kevin A Strauss, Michelle A Farrar Mbbs, Francesco Muntoni   +2 more
exaly  

Abordaje Interdisciplinario en la Atrofia Muscular Espinal.

2023
openaire   +1 more source

Base editing rescue of spinal muscular atrophy in cells and in mice

Science, 2023
, Zaneta Matuszek
exaly  

Atrofia muscular espinal tipo 1 con distrés respiratorio

Revista de Neurología, 2013
Silvia Calderón Rodríguez, Verónica Cantarín Extremera, María Ángeles García Teresa, Montserrat Nieto Moro, Juan Casado Flores, Fernando Martín del Valle, Anna Duat Rodríguez   +6 more
openaire   +1 more source

Wearable full-body motion tracking of activities of daily living predicts disease trajectory in Duchenne muscular dystrophy

Nature Medicine, 2023
Valeria Ricotti, Thomas Voit, Aldo A Faisal   +2 more
exaly  

Pharmacological strategies for muscular dystrophy

Nature Reviews Drug Discovery, 2003
Tejvir S Khurana, Kay E Davies
exaly  

Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study

Lancet, The, 2018
Craig M Mcdonald, Erik K Henricson, Paula R Clemens   +2 more
exaly  

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

Lancet, The, 2017
Craig M Mcdonald, Craig Campbell, Richard S Finkel   +2 more
exaly  

Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial

Lancet, The, 2015
Maria Grazia D'Angelo, Richard S Finkel, Craig M Mcdonald   +2 more
exaly  

Anestesia epidural en paciente con atrofia muscular espinal tipo kugelberg-welander

Revista Española de Anestesiología y Reanimación, 2008
P. Fernández Garijo, M.L. Gómez Martínez, N. Hernández Martín, J.I. Gómez Herreras   +3 more
openaire   +1 more source