Results 21 to 30 of about 106,524 (297)

Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy

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, P. Clemens, Seth Perlman, E. Smith, I. Horrocks, R. Finkel, J. Mah, N. Deconinck, N. Goemans, J. Haberlova, Volker Straub, L. Mengle-Gaw, Benjamin D. Schwartz, A. Harper, P. Shieh, L. De Waele, D. Castro, Michelle L. Yang, M. Ryan, C. McDonald, M. Tulinius, R. Webster, H. McMillan, N. Kuntz, Vashmi K Rao, G. Baranello, S. Spinty, A. Childs, A. M. Sbrocchi, K. Selby, M. Monduy, Y. Nevo, J. Vilchez-Padilla, Andrés Nascimento-Osorio, E. Niks, I. D. de Groot, M. Katsalouli, M. James, J. N. van den Anker, J. Damsker, A. Ahmet, L. Ward, M. Jaros, Phil Shale, U. Dang, E. Hoffman   +45 more
semanticscholar   +1 more source

Modeling Patient-Specific Muscular Dystrophy Phenotypes and Therapeutic Responses in Reprogrammed Myotubes Engineered on Micromolded Gelatin Hydrogels

Frontiers in Cell and Developmental Biology, 2022
In vitro models of patient-derived muscle allow for more efficient development of genetic medicines for the muscular dystrophies, which often present mutation-specific pathologies.
Florian Barthélémy, Florian Barthélémy, Jeffrey W. Santoso, Laura Rabichow, Laura Rabichow, Rongcheng Jin, Isaiah Little, Isaiah Little, Stanley F. Nelson, Stanley F. Nelson, Stanley F. Nelson, Megan L. McCain, Megan L. McCain, M. Carrie Miceli, M. Carrie Miceli   +14 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, P. Clemens, M. Guglieri, E. Smith, R. Finkel, M. Tulinius, Y. Nevo, M. Ryan, R. Webster, D. Castro, N. Kuntz, C. McDonald, J. Damsker, Benjamin D. Schwartz, L. Mengle-Gaw, S. Jackowski, Georgia Stimpson, D. Ridout, V. Ayyar-Gupta, G. Baranello, A. Manzur, F. Muntoni, H. Gordish-Dressman, M. Leinonen, L. Ward, E. Hoffman, U. Dang   +26 more
semanticscholar   +1 more source

Cytokines and chemokines as regulators of skeletal muscle inflammation: presenting the case of Duchenne muscular dystrophy [PDF]

, 2013
Duchenne muscular dystrophy is a severe inherited muscle disease that affects 1 in 3500 boys worldwide. Infiltration of skeletal muscle by inflammatory cells is an important facet of disease pathophysiology and is strongly associated with disease ...
De Bleecker, Jan, De Paepe, Boel
core   +3 more sources

Progressive muscular dystrophy (Duchenne type) (case report).

Paediatrica Indonesiana, 2019
Clinical findings of two brothers suffering from progressive muscular dystrophy pseudohypertrophic type according to Duchenne are reported. Literatures dealing with its clinical classification, biochemical disturbances, hypotheses of the pathogenesis ...
S. Winarno, Indrawarman, S. Waloejo, L. Kristanti   +3 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, A. C. Chai, H. Li, C. Rodriguez-Caycedo, E. Sánchez-Ortiz, A. Atmanli, A. Mireault, N. Liu, R. Bassel-Duby, E. Olson   +9 more
semanticscholar   +1 more source

Repurposing Dantrolene for Long-Term Combination Therapy to Potentiate Antisense-Mediated DMD Exon Skipping in the mdx Mouse

Molecular Therapy: Nucleic Acids, 2018
Duchenne muscular dystrophy (DMD) is caused by mutations in DMD, resulting in loss of dystrophin, which is essential to muscle health. DMD “exon skipping” uses anti-sense oligo-nucleotides (AONs) to force specific exon exclusion during mRNA processing to
Derek W. Wang, Ekaterina I. Mokhonova, Genevieve C. Kendall, Diana Becerra, Yalda B. Naeini, Rita M. Cantor, Melissa J. Spencer, Stanley F. Nelson, M. Carrie Miceli   +8 more
doaj   +1 more source

The quality of life in boys with Duchenne muscular dystrophy [PDF]

, 2016
We conducted a study to evaluate the quality of life in boys with Duchenne muscular dystrophy aged 8–18 years, compared with that in matched healthy controls. A total of 85 boys with Duchenne muscular dystrophy aged 8–18 years and 136 age, sex and living
Ashrafi, M.R., Azizi Malamiri, R., Fathi, M.R., Heidari, M., Hosseini, S.A., Mohammadi, M., Qorbani, M., Salehi, S., Shahrokhi, A., Shervin Badv, R., Zamani, G.   +10 more
core   +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, M. Hill, M. Guglieri, M. Crowther, K. Abrams   +4 more
semanticscholar   +1 more source

Skeletal Muscle Membrane Permeability Markers Derived From ³¹P-MRS May Reflect Disease Activity in Becker Muscular Dystrophy. [PDF]

NMR Biomed
Biomarkers for muscle disease activity are needed for trials in Becker muscular dystrophy (BMD). We investigated magnesium (Mg2+), phosphodiesters (PDE) and pH from 31P‐MRS; and membrane permeability from random permeable barrier model (RBPM) diffusion as candidates, studying ‘preserved’ and ‘progressing’ muscles in patients with BMD versus controls ...
Schrama EJ, Hooijmans MT, van de Velde NM, Niks EH, Kan HE, Cameron D.   +5 more
europepmc   +2 more sources