Early safety and efficacy data from a Phase 1 trial suggest that Taiho Pharmaceutical’s investigational compound TAS-205 may hold the potential to treat patients with Duchenne’s muscular dystrophy (DMD). The results were reported in the study, “A phase I…
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Like many mothers of boys with muscular dystrophy, Deborah Hall hasn’t had an easy life. Hall, the daughter of a chemical engineer and a stay-at-home mom, was one of six siblings growing up in Boaz, West Virginia. When her younger brother, John, was 3 years old, doctors diagnosed him with…
Body mass index (BMI) and blood pressure are modifiable factors that could help to slow heart deterioration in patients with Duchenne muscular dystrophy (DMD), according to a retrospective study. The study, “Increased Blood Pressure and Body Mass Index as Potential Modifiable Factors in The Progression of Myocardial Dysfunction…
Metformin, a diabetes treatment, eased disease progression and improved muscle function in female — and, to a lesser extent, in male — mice altered to have congenital muscular dystrophy with laminin α2 chain-deficiency (LAMA2-CMD), a study reports. Its researchers also reported that this type of congenital muscular dystrophy — the second…
Duchenne UK announced that it has awarded a sizable grant to Evox Therapeutics to advance in early testing a way of using gene therapy to deliver — without a virus as a transport agent — the dystrophin gene or shorter versions of it to people with Duchenne muscular dystrophy. The group’s…
This month’s Democratic takeover of the U.S. House of Representatives following the Nov. 6 midterm elections could be good news for Americans with rare diseases, says the National Organization for Rare Disorders (NORD), which represents 280 patient advocacy organizations. “The entire community advocated against the repeal of the Affordable Care…
Catabasis Pharmaceuticals will collaborate with the University of Texas Southwestern to investigate potential benefits of edasalonexent on heart function in Duchenne and Becker muscular dystrophies. After the promising results of Catabasis’ Phase 1/2 MoveDMD trial (NCT02439216) and its open-label extension, edasalonexent is now being…
CureDuchenne and Avidity Biosciences will host a webinar Friday to brief the Duchenne muscular dystrophy (DMD) community on Avidity’s preclinical development of DMD therapy candidates. The webinar will take place at 4 p.m. EST and will feature Debra Miller, CureDuchenne’s founder and CEO, and Arthur A. Levin,…
Extracellular RNAs found in urine may be able to serve as non-invasive biomarkers of how patients with muscular dystrophy — especially those with an adult-onset and the more common Duchenne MD form — are responding to therapy, a study reports. Urine-sample tests for these RNAs may also lessen the need…
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Phase 3 Trial of Edasalonexent in Boys with DMD and Any Mutation Enrolling in US, Catabasis Reports
A pivotal Phase 3 trial of edasalonexent, a potential oral disease-modifying treatment for Duchenne muscular dystrophy (DMD) regardless of mutation type, is enrolling boys ages 4 to 7 in the U.S. and will soon begin with those in Australia, Canada, Europe, and Israel. “We named the trial ‘Polaris’ because it…
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