The U.S. Food and Drug Administration (FDA) yesterday announced the approval of the corticosteroid drug deflazacort (under the trade name Emflaza) for the treatment of Duchenne muscular dystrophy (DMD).
An update on the current therapeutic approaches being developed to treat Duchenne muscular dystrophy (DMD) has been published in the journal Pharmacotherapy.
Researchers suggest a mutation in the INPP5K gene caused a new type of congenital muscular dystrophy. Individuals affected by the unique mutation also have a short stature, intellectual disabilities, and cataracts. The study, “Mutations in INPP5K Cause a Form of Congenital Muscular Dystrophy Overlapping Marinesco-Sjögren Syndrome and Dystroglycanopathy,” was a…
Two non-invasive strategies for measuring muscle function and its properties, electromechanical delay and ultrasound shear wave elastography, may help detect muscle impairments and assist in monitoring Duchenne muscular dystrophy (DMD) patients’ responses to therapy. The study “Effects of Duchenne muscular dystrophy on muscle stiffness and response to electrically-induced muscle…
Gene variations that decrease the production of a certain protein appear to reduce muscle strength in patients with Duchenne muscular dystrophy (DMD) but improve their disease progression, according to a study. The research, “Evidence For ACTN3 As A Genetic Modifier Of Duchenne Muscular Dystrophy,” was published in the journal…
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…
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