Results 11 to 20 of about 71,356 (167)

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, Liesbeth 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

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

Improved Muscle Function in Duchenne Muscular Dystrophy through L-Arginine and Metformin: An Investigator-Initiated, Open-Label, Single-Center, Proof-Of-Concept-Study. [PDF]

PLoS ONE, 2016
Altered neuronal nitric oxide synthase function in Duchenne muscular dystrophy leads to impaired mitochondrial function which is thought to be one cause of muscle damage in this disease.
Patricia Hafner, Ulrike Bonati, Beat Erne, Maurice Schmid, Daniela Rubino, Urs Pohlman, Thomas Peters, Erich Rutz, Stephan Frank, Cornelia Neuhaus, Stefanie Deuster, Monika Gloor, Oliver Bieri, Arne Fischmann, Michael Sinnreich, Nuri Gueven, Dirk Fischer   +16 more
doaj   +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

Gait Event Detection and Travel Distance Using Waist-Worn Accelerometers across a Range of Speeds: Automated Approach [PDF]

Sensors. 2024; 24(4):1155, 2023
Estimation of temporospatial clinical features of gait (CFs), such as step count and length, step duration, step frequency, gait speed, and distance traveled, is an important component of community-based mobility evaluation using wearable accelerometers.
arxiv   +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

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

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

Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double‐blind, placebo‐controlled, dose‐titration trial

Muscle and Nerve, 2021
Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene resulting in the absence of dystrophin. Casimersen is a phosphorodiamidate morpholino oligomer designed to bypass frameshift DMD mutations and produce internally truncated, yet ...
K. Wagner, N. Kuntz, E. Koenig, L. East, S. Upadhyay, B. Han, P. Shieh   +6 more
semanticscholar   +1 more source

Beyond MeSH: Fine-Grained Semantic Indexing of Biomedical Literature based on Weak Supervision [PDF]

Information Processing and Management 57 (2020) 102282, 2020
In this work, we propose a method for the automated refinement of subject annotations in biomedical literature at the level of concepts. Semantic indexing and search of biomedical articles in MEDLINE/PubMed are based on semantic subject annotations with MeSH descriptors that may correspond to several related but distinct biomedical concepts.
arxiv   +1 more source