Catabasis Pharmaceuticals reported mixed topline data from Part B of the MoveDMD trial evaluating the safety and efficacy of CAT-1004 (edasalonexent) in treating Duchenne muscular dystrophy (DMD) patients. MoveDMD is a three-part, multisite Phase 1/2 trial (NCT02439216) evaluating the efficacy, safety, and the pharmacodynamics (PD) and pharmacokinetics (PK),…
The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Resolaris as a potential treatment for limb girdle muscular dystrophy 2B (LGMD2B). The FDA also removed its partial clinical hold on a dosing ceiling for Resolaris in clinical trials, the drug’s developer, aTyr Pharma, announced in press…
Appropriate health care in Duchenne muscular dystrophy (DMD) can markedly increase quality of sleep and quality of life according to a study presenting the case of two women who are manifesting carriers of the disease.
A researcher at the University of Texas Southwestern Medical Center has been awarded a $250,000 grant to study CRISPR/Cas9 technology‘s potential to treat Duchenne muscular dystrophy (DMD). CRISPR/Cas9 allows researchers to add, remove, or change sections of the DNA sequence. Parent Project Muscular Dystrophy (PPMD) will give the…
Bone fragility is a serious consequence of long-term corticosteroid treatment — the main option for Duchenne muscular dystrophy (DMD) — but an analysis found that there are no high-quality studies on how to improve patients’ bone health. The analysis, “Interventions to prevent and treat corticosteroid-induced osteoporosis and prevent…
People with Duchenne muscular dystrophy have a lower heart rate variability than healthy people — an abnormality that worsened after completing a computer task. Since low heart rate variability has been reported as a risk factor for heart failure, the finding indicates that more research is needed to better understand the…
The binding of two proteins called titin and α-actinin ensures that muscles are not ripped apart when they are forced to stretch, according to new research. Scientists believe the identification and study of this protein interaction may offer new possibilities for the treatment of diseases such as muscular dystrophy. The…
Engagement in physical activity can increase the sense of self-worth and belonging to a group, and can help form peer relationships in children with Duchenne muscular dystrophy (MD), according to a study published in the Journal of Child Neurology.
A Duchenne muscular dystrophy (DMD) patient who is now in his 50s gained the notice of researchers, who say his case could illustrate the benefits of noninvasive mechanical ventilation in patients with well-maintained cardiac function. It is the first report of a patient living into his sixth decade. The report, “New Survival…
Yale New Haven Children’s Hospital’s (YNHCH) Muscular Dystrophy Program has been named a Certified Duchenne Care Center by Parent Project Muscular Dystrophy (PPMD), a nonprofit organization dedicated to ending Duchenne muscular dystrophy (DMD). PPMD’s Certified Duchenne Care Center Program, which was started in 2014, supports standardized, comprehensive…
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