Results 11 to 20 of about 66,813 (317)
Journal of the American Medical Association (JAMA), 2022 Importance Corticosteroids improve strength and function in boys with Duchenne muscular dystrophy. However, there is uncertainty regarding the optimum regimen and dosage.
M. Guglieri +62 more
semanticscholar +1 more source
Precise correction of Duchenne muscular dystrophy exon deletion mutations by base and prime editing
Science Advances, 2021 Base and prime genome editing correct Duchenne muscular dystrophy mutations to restore dystrophin in mice and human cells. Duchenne muscular dystrophy (DMD) is a fatal muscle disease caused by the lack of dystrophin, which maintains muscle membrane ...
F. Chemello +9 more
semanticscholar +1 more source
Update of the Brazilian consensus recommendations on Duchenne muscular dystrophy
Arquivos de Neuro-Psiquiatria, 2023 In the last few decades, there have been considerable improvements in the diagnosis and care of Duchenne muscular dystrophy (DMD), the most common childhood muscular dystrophy. International guidelines have been published and recently reviewed.
Alexandra Prufer de Queiroz Campos Araujo +21 more
doaj +1 more source
Molecules, 2021 Duchenne muscular dystrophy (DMD) is a progressive fatal neuromuscular disorder with no cure. Therapies to restore dystrophin deficiency have been approved in some jurisdictions but long-term effectiveness is yet to be established.
Keryn G. Woodman +3 more
doaj +1 more source
JAMA Neurology, 2022 This randomized clinical trial investigates the efficacy and safety of partial receptor agonist vamorolone compared with placebo and prednisone in boys with Duchenne muscular dystrophy.
M. Guglieri +45 more
semanticscholar +1 more source
On the relevance of prognostic information for clinical trials: A theoretical quantification
Biometrical Journal, Volume 65, Issue 1, January 2023., 2023 Abstract The question of how individual patient data from cohort studies or historical clinical trials can be leveraged for designing more powerful, or smaller yet equally powerful, clinical trials becomes increasingly important in the era of digitalization.
Sandra Siegfried +2 more
wiley +1 more source
Penalized regression calibration: a method for the prediction of survival outcomes using complex longitudinal and high-dimensional data [PDF]
Statistics in Medicine (2021), 2021 Longitudinal and high-dimensional measurements have become increasingly common in biomedical research. However, methods to predict survival outcomes using covariates that are both longitudinal and high-dimensional are currently missing. In this article, we propose penalized regression calibration (PRC), a method that can be employed to predict survival
arxiv +1 more source
Life Expectancy in Duchenne Muscular Dystrophy
Neurology, 2021 Background and Objectives Duchenne muscular dystrophy (DMD) is a rare progressive disease that is often diagnosed in early childhood and leads to considerably reduced life expectancy; because of its rarity, research literature and patient numbers are ...
J. Broomfield +4 more
semanticscholar +1 more source
The importance of direct genetic testing for determining female carriers of the mutation in dystrophinopathies [PDF]
Vojnosanitetski Pregled, 2023 Background/Aim. Duchenne muscular dystrophy (MD) and Becker MD are caused by mutations in the gene for dystrophin (DMD). They are X chromosome-linked recessive diseases where males are affected, and females are healthy carriers of the mutation in most ...
Maksić Jasmina +7 more
doaj +1 more source
Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy
JAMA Network Open, 2022 This nonrandomized controlled trial examines efficacy of vamorolone treatment for Duchenne muscular dystrophy among boys compared with glucocorticoid treatment.
J. Mah +26 more
semanticscholar +1 more source