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…
When your child starts school you may need some help settling in and preparing the school and teachers for meeting your child’s needs. The Muscular Dystrophy Association (MDA) offers a range of educational resources designed to help children with muscular dystrophy and spinal muscular atrophy (SMA) get the most…
https://www.youtube.com/watch?v=KW4WvLd5NqY This video from the World Duchenne Awareness Day shares some of the early symptoms of Duchenne muscular dystrophy. It’s estimated that one in 3500 boys will be born with Duchenne muscular dystrophy but most won’t present any symptoms of the disease until they are two or three years old.
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…
Most three-year-olds only get to watch marathons from the sidelines or on television, but not Levi Gagner. The Rare Disease Report revealed that Levi, along with his dad Kyle, completed the Rock ‘n’ Roll half marathon last weekend. The Pheonix, Arizona event is the third race the two have competed…
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…
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