Duchenne muscular dystrophy (DMD) is a rare hereditary disease that is associated with progressive muscle wasting. The ...
Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...
European regulators are to review Sarepta Therapeutics’ Duchenne ... muscular dystrophy (DMD) patients amenable to exon 51 skipping - about 13% of the population with the rare muscle-wasting ...
Evan McMenamin said his brother Travis, "never lets it get him down" despite the "incredible challenges" he faces ...
Santhera Pharmaceuticals will launch its new drug for Duchenne muscular dystrophy in the UK within ... that has been shown in trials to slow down muscle degeneration and prolong the ability ...
Peer ReviewDownload a summary of the editorial decision process including editorial decision letters, reviewer comments and author responses to feedback. Immune responses play an integral role in ...
Limb-girdle muscular dystrophy type 2D (LGMD2D/R3) is a rare genetic disorder caused by mutations in the SGCA gene, leading to defective folding and the loss of functional α-sarcoglycan, with ...
Muscle biopsy specimens were available from 119 myositis patients including 39 with DM (11 anti-Mi2-positive, 12 anti-NXP2-positive, 11 anti-TIF1γ-positive and 5 anti-MDA5-positive), 49 with IMNM (9 ...
Chair and Professor of Medicinal Chemistry, Joseph P. & Shirley Shipman Buckley Endowed Professor of Drug Discovery Synthetic medicinal chemistry, structure-based design, natural product chemistry ...
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