Limb-girdle muscular dystrophy (LGMD) may be caused by previously unidentified TTN gene variants that are also associated with heart muscle disease, according to a study. Findings of the study, “Investigation of TTN variants in patients with Limb-Girdle Muscular Dystrophy identifies novel Titinopathies,” were presented by…
Very young boys with Duchenne muscular dystrophy (DMD) have measurable differences in motor skills, which could have implications for the early detection of DMD and for evaluating how treatments affect their development as opposed to the natural course of the disease, a study shows. Findings of the study, “…
RG6206 (BMS-986089), a blocker of muscle growth-inhibitor myostatin, is safe to use in boys with Duchenne muscular dystrophy (DMD), results from a Phase 1b/2 trial show. A Phase 2/3 trial of the drug (NCT03039686) is actively recruiting patients. Moreover, the results showed that the compound was able to reduce…
The investigational gene therapy GALGT2 for Duchenne muscular dystrophy (DMD) was found to be safe in a preclinical study conducted at the Research Institute at the Nationwide Children’s Hospital, the researchers said. The study was funded by a $100,000 grant awarded by Parent Project Muscular…
A protein called epidermal growth factor (EGF) can help preserve muscle strength and increase muscle regeneration in a mouse model of Duchenne muscular dystrophy (DMD), a finding that may pave the way for new treatment strategies for DMD, researchers said. The study, “EGFR-Aurka Signaling Rescues Polarity and…
Cumbersome security procedures, rising airfares, and shrinking legroom have made commercial air travel difficult enough these days — even for healthy passengers. Imagine how much harder it is for patients with rare diseases who must get to doctors’ appointments or clinical trials that are hundreds of miles away from home.
Merosin-deficient muscular dystrophy, a type of congenital MD, might be diagnosed before birth using immunohistochemistry, an easy and quick lab test that looks for specific markers in cells from a mother’s placenta, according to a small study of pregnant women whose children are at risk for this disease. A misdiagnosis, however,…
A signaling pathway determines whether muscle stem cells differentiate and mature into working muscle cells, an early study in mice found. Targeting this pathway and its two proteins — Fam3a and Stat3, which works to stimulate Fam3a — may treat diseases or illnesses of muscles, including muscular dystrophies. Getting these stem cells to…
Fulcrum Therapeutics announced it has acquired global rights to develop and potentially market losmapimod, an investigational treatment for facioscapulohumeral muscular dystrophy (FSHD), and that it plans to open a Phase 2 clinical trial testing the compound’s safety and efficacy this year. The compound was initially developed by GlaxoSmithKline (GSK) for…
Promising safety and tolerability data from a Phase 1 trial on suvodirsen (WVE-210201), Wave Life Sciences‘ investigational exon-skipping therapy for boys with Duchenne muscular dystrophy (DMD), opens the door for the launch of new Phase 2/3 clinical trials later this year. Final results from the trial (NCT03508947) were…
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