A single gene therapy that silences the mutation responsible for oculopharyngeal muscular dystrophy (OPMD) and replaces the mutated gene with a normal one may advance into human studies in the second half of 2018. Benitec Biopharma started its OPMD program in 2014 and now announced their clinical candidate BB-301…
Parent Project Muscular Dystrophy (PPMD) will host a one-hour webinar at 2 p.m. Eastern Time on Wednesday, Sept. 6, that will focus on an upcoming clinical trial exploring gene therapy for Duchenne muscular dystrophy. The webinar will be led by Dr. Jerry Mendell, who, together with fellow researcher Dr. Louise…
With technology at our fingertips, being unplugged and off grid can seem like something that exists only in a fiction novel. New smartphones come out each year, cars have built-in wifi, and music and television can be accessed from our almost anywhere. Just like everything in life, technology has its pros…
In what could be the first step on the long road to finding a potential cure, researchers have found that aberrations in RNA processing are the major cause of congenital myotonic dystrophy (CDM). The study “Disrupted prenatal RNA processing and myogenesis in…
Treadmill training using a voluntary-driven exoskeleton might be a safe approach to improve movement and exercise capacity in patients with limb-girdle muscular dystrophy, according to a small study by researchers at Ruhr University Bochum in Germany. The study, “Treadmill Training with HAL Exoskeleton — A Novel Approach for Symptomatic…
MRIs can pick up subtle muscle changes that standard tests cannot in slowly progressing disorders such as facioscapulohumeral muscular dystrophy, or FSHD, a study reports. The research in the journal PLoS ONE was titled “Long-term follow-up of MRI changes in thigh muscles of patients with Facioscapulohumeral dystrophy: A quantitative…
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…
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