Results 281 to 290 of about 71,938 (313)

PREDNISONE IN DUCHENNE MUSCULAR DYSTROPHY

The Lancet, 1974
Abstract Fourteen patients with typical Duchenne muscular dystrophy were treated with prednisone for up to 28 months. Thirteen patients showed improvement in motor power and muscular activities while on prednisone. In eight of these, the improvement has been maintained for up to 28 months, while in five others deterioration has occurred while on ...
D B, Drachman, K V, Toyka, E, Myer
openaire   +2 more sources

Pharmacotherapy of Duchenne Muscular Dystrophy

2019
Drug development and pharmacotherapy of rare pediatric diseases have significantly expanded over the last decade, in part due to incentives and financial support provided by governments, regulators, and nonprofit foundations. Duchenne muscular dystrophy (DMD) is among the most common rare pediatric disorders, and clinical trials of therapeutic ...
openaire   +2 more sources

Survival in Duchenne muscular dystrophy.

Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology, 2012
To determine the survival in a population of German patients with Duchenne muscular dystrophy.Information about 94 patients born between 1970 and 1980 was obtained by telephone interviews and questionnaires. In addition to age of death or actual age during the investigation, data concerning clinical course and medical interventions were collected.67 ...
Rall, Susanne, Grimm, Tiemo
openaire   +2 more sources

Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial

Nature Medicine, 2022
Kevin A Strauss, Michelle A Farrar Mbbs
exaly  

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  

Carnitine in duchenne muscular dystrophy

Clinica Chimica Acta, 1979
T, Kobayashi, N, Shinnoh, S, Mawatari
openaire   +2 more sources

Therapeutic approaches for Duchenne muscular dystrophy

Nature Reviews Drug Discovery, 2023
Thomas C Roberts, Kay E Davies
exaly  

Spinal muscular atrophy

Nature Reviews Disease Primers, 2022
Francesco Muntoni, Richard S Finkel
exaly  

Steroids in Duchenne muscular dystrophy

Neuromuscular Disorders, 2013
V, Ricotti, D A, Ridout, F, Muntoni
openaire   +2 more sources

Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy

Nature Medicine, 2020
Alessandra Moretti, Daniel Sinnecker, Shicheng Sun   +2 more
exaly