News

The chemical structure of a drug can make the entire difference between a success and a failure in drug development. Of two drugs that used the same approach to treat Duchenne muscular dystrophy — Exondys 51 (eteplirsen) and Kyndrisa (drisapersen) — only one became approved. Researchers from The Ohio…

Researchers have found Duchenne muscular dystrophy (DMD) patients with heart disease have very high levels of ST2, an interleukin 1 receptor-like 1 protein. That makes this molecule a potential biomarker for cardiac disease in such patients. The study by scientists at Children’s National Health System in Washington, D.C., “Interleukin 1…

Researchers, using a mouse model of Duchenne muscular dystrophy (DMD), have discovered that a specific protein, dystrophin, plays an essential role in myelination during postnatal brain development. The protein dystrophin plays a major role in DMD. Mutations in the dystrophin gene are a major contributing factor for the development of…

Successful results of Translarna (ataluren) and Emflaza (deflazacort) therapies for Duchenne muscular dystrophy (DMD) are highlighted in the 2017 second quarter report by PTC Therapeutics. Orally-administered Translarna is for Duchenne patients to reduce the effect of nonsense mutations in the DMD gene that lead to a lack of…

Synergy HomeCare and the nonprofit Muscular Dystrophy Association (MDA) have teamed up in a national partnership to improve quality of life and give more independence to people with neuromuscular disorders. The accord aims to develop a new care guide for newly diagnosed patients and their primary caregivers. The guide will offer…

Many patients with Charcot-Marie-Tooth (CMT) disease, a subtype of muscular dystrophy, undergo surgeries to correct foot deformities. Yet a new study finds that surgeons vary significantly in their choice of procedures for the same operation. The study, “Prevalence and orthopedic management of foot and ankle deformities in Charcot Marie Tooth disease,”…

Capricor Therapeutics recently shared an update of the company’s quarterly financial results and operational highlights, including positive results for the ongoing HOPE study with Duchenne muscular dystrophy (DMD) patients. The Phase I/II HOPE study (NCT02485938) is assessing the safety and tolerability of investigational…

The American Heart Association (AHA) has awarded its distinguished Scientist Development Grant to a University at Albany researcher for the development of new therapies to treat Duchenne muscular dystrophy (DMD). Bijan K. Dey, PhD, is a faculty researcher at the university’s RNA Institute and is working on a class of…

Cardiff University scientists, partnering with the biotech company PerkinElmer, have developed a diagnostic kit to screen for Duchenne muscular dystrophy (DMD) in newborns using dried blood spots, according to researchers. The study, “Characterization of a Blood Spot Creatine Kinase Skeletal Muscle Isoform Immunoassay for High-Throughput Newborn Screening of Duchenne…

The investigational therapy MA-0211, also called MTB-1, a potential treatment for Duchenne Muscular Dystrophy (DMD), was approved for a Phase I clinical trial, developer Mitobridge announced. The trial will evaluate the therapy’s safety and tolerability and determine its pharmacokinetics and pharmacodynamics profile, or its behavior in the body and mechanism…