Results 91 to 100 of about 169,015 (342)

Barriers to diverse clinical trial participation in Duchenne muscular dystrophy: Engaging Hispanic/Latina caregivers and health professionals

Orphanet Journal of Rare Diseases
Background Despite the increasing availability of clinical trials in Duchenne muscular dystrophy, racial/ethnic minorities and other populations facing health disparities remain underrepresented in clinical trials evaluating products for Duchenne.
Norah L. Crossnohere, Nicola B. Campoamor, Eric Camino, Erin Dresnick, Daphne Oluwaseun Martschenko, Viana Rodrigues, Susan Apkon, Alexis Hazlett, Dhruv Mittur, Priscilla E. Rodriguez, John F. P. Bridges, Niki Armstrong   +11 more
doaj   +1 more source

Chart validation of an algorithm for identifying hereditary progressive muscular dystrophy in healthcare claims

BMC Medical Research Methodology, 2019
Background Muscular dystrophies (MDs) are a group of inherited conditions characterized by progressive muscle degeneration and weakness. The rarity and heterogeneity of the population with MD have hindered therapeutic developments as well as ...
Xiaoxue Chen, Abiy Agiro, Ann S. Martin, Ann M. Lucas, Kevin Haynes   +4 more
doaj   +1 more source

Osteopontin ablation ameliorates muscular dystrophy by shifting macrophages to a pro-regenerative phenotype. [PDF]

, 2016
In the degenerative disease Duchenne muscular dystrophy, inflammatory cells enter muscles in response to repetitive muscle damage. Immune factors are required for muscle regeneration, but chronic inflammation creates a profibrotic milieu that exacerbates
Barton, Elisabeth R, Capote, Joana, Kramerova, Irina, Martinez, Leonel, Miceli, M Carrie, Spencer, Melissa J, Sweeney, H Lee, Vetrone, Sylvia   +7 more
core   +2 more sources

Techno‐Economic Analysis of Membrane‐Based Purification Platforms for AAV Vector Production

Biotechnology and Bioengineering, EarlyView.
ABSTRACT Technologies for large‐scale manufacturing of viral vectors for gene therapies, such as tangential flow filtration and membrane chromatography, are under development. In these early stages of process development, techno‐economic analyses are useful for identifying membrane properties yielding the greatest impact on process performance. In this
Juan J. Romero, Eleanor W. Jenkins, Jacob I. Monroe, S. Ranil Wickramasinghe, Xianghong Qian, Dibakar Bhattacharyya, Scott M. Husson   +6 more
wiley   +1 more source

Adjuvant pharmacological strategies for the musculoskeletal system during long‐term space missions

British Journal of Clinical Pharmacology, EarlyView., 2023
Abstract Despite 2 h of daily exercise training, muscle wasting and bone loss are still present after 6‐month missions to the international space station. Some crew members lose bone much faster than others. In preparation for missions to the Moon and Mars, space agencies are therefore reviewing their countermeasure portfolios.
Friederike Thomasius, Dominik Pesta, Jörn Rittweger   +2 more
wiley   +1 more source

MATR3 is an endogenous inhibitor of DUX4 in FSHD muscular dystrophy

Cell Reports, 2023
Summary: Facioscapulohumeral muscular dystrophy (FSHD) is one of the most common neuromuscular disorders and has no cure. Due to an unknown molecular mechanism, FSHD displays overlapping manifestations with the neurodegenerative disease amyotrophic ...
Valeria Runfola, Roberto Giambruno, Claudia Caronni, Maria Pannese, Annapaola Andolfo, Davide Gabellini   +5 more
doaj  

Overexpression of Mutant FKRP Restores Functional Glycosylation and Improves Dystrophic Phenotype in FKRP Mutant Mice

Molecular Therapy: Nucleic Acids, 2018
Autosomal recessive homozygous or compound heterozygous mutations in FKRP result in forms of muscular dystrophy-dystroglycanopathy varying in age of onset, clinical presentation, and disease progression, ranging from the severe Walker-Warburg, type A,5 ...
Jason D. Tucker, Pei J. Lu, Xiao Xiao, Qi L. Lu   +3 more
doaj   +1 more source

The Muscular Dystrophy Association

, 2017
Muscular dystrophy (MD) is a social problem because the people affected by it do not get to live a normal life.https://digitalscholarship.unlv.edu/educ_sys_202/1016/thumbnail ...
Ortiz, Natasha
core  

The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development [PDF]

, 2015
Despite multiple publications on potential therapies for neuromuscular diseases (NMD) in cell and animal models only a handful reach clinical trials. The ability to prioritise drug development according to objective criteria is particularly critical in ...
Allen, Hugh, Bourke, John, Burghes, Arthur, Bushby, Kate, Buyse, Gunnar, Caizergues, Didier, Catlin, Nick, Clemens, Paula, Cnaan, Avital, Comi, Giacomo, Connor, Edward, Csimma, Cristina, Day, Simon, de Luca, Annamaria, de Montleau, Béatrice, de Visser, Marianne, Dittrich, Sven, Dubrosky, Alberto, Eagle, Michelle, Finkel, Richard, Fishbeck, Kenneth, Flanigan, Kevin M., Furlong, Patricia, Grounds, Miranda, Hauschke, Dieter, Heslop, Emma, Hesterlee, Sharon, Hoffman, Eric, Irwin, Joseph, Jarecki, Jill, Kaufmann, Petra, Kelly, Michael, Korinthenberg, Rudolf, Laforêt, Pascal, Larkindale, Jane, Lovering, Richard, Mayer, Henry, McCall, John, McDonald, Robert, McNally, Elizabeth, McNeil, Dawn Elizabeth, McNeil, Elizabeth, Mendell, Jerry, Miller, Debra, Nagaraju, Kanneboyina, North, Kathryn, Ouillade, Marie-Christine, Straub, Volker, Wagner, Kathryn R., Wells, Dominic J.   +49 more
core   +4 more sources

Docking and Molecular Dynamics Simulation‐Based Analysis of Advanced Small‐Molecule Kinase Inhibitors Identified pre‐let‐7 miRNA Binders

ChemBioChem, Accepted Article.
RNAs play crucial roles in various cellular actions, and the uncontrolled expression or improper folding of RNAs is a cause of many diseases. Certain oncogenic phenotypes stem from the overexpression of regulatory microRNAs that contain secondary structural elements.
Soma Roy, Yang Liu, Peng Wu
wiley   +1 more source