News

Taking care of patients with Duchenne muscular dystrophy (DMD) can have significant effects on caregivers, leading to a lower health‐related quality of life, poor sleep quality, depression, pain, stress, sexual dysfunction, and lower self‐esteem, a review study reports. Caregiving can also have a considerable negative impact on work life…

Researchers at The University of Western Australia (UWA) have been awarded close to $490,000 from two organizations to explore the potential use of an amino acid called taurine as a treatment for Duchenne muscular dystrophy (DMD). Duchenne UK granted £273,648 (approximately $356,515) to support the study, while Save our Sons…

The digital health firm ZappRx is working with CureDuchenne to provide faster and more efficient delivery of treatments for Duchenne muscular dystrophy to patients starting next year. ZappRx streamlines the often-complicated processes involved in ordering specialty medications by collecting and storing required information from pharmacies, insurance companies and medical providers.

CureDuchenne, a nonprofit advocacy group that supports and promotes research and better care and for people with Duchenne muscular dystrophy (DMD), has announced the launch of a three-year partnership with Baebies in which the company will add DMD to its expanded newborn screening service. The goal is to make…

A new investigational therapy called GsMTx4, developed by researchers at the University of Buffalo and based on a molecule found in tarantula venom, is able to prevent the loss of muscle mass and muscle injury in a mouse model of advanced Duchenne muscular dystrophy (DMD). These findings were published in…

Sarepta Therapeutics‘ microdystrophin gene therapy enhanced dystrophin protein expression in the muscles of four boys with Duchenne muscular dystrophy (DMD) enrolled in a Phase 1/2 trial. More importantly, the gene therapy improved the boys’ functional performance. “The encouraging results that we previously saw and reinforced in the fourth…

Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire fraud while heading San Diego-based Retrophin. As founder and CEO of another company (then known as Turing Pharmaceuticals), in late 2015 Shkreli bought the rights…

One of New England’s top experts in Duchenne muscular dystrophy will give the keynote address at a conference next month dedicated to finding a cure for the neuromuscular disease. Brenda Wong, MD, is the director of the Duchenne program at University of Massachusetts Medical in Worcester, about a 45-minute…

The Greiner family recently held its 10th Miles for Matthew fundraising event for Parent Project Muscular Dystrophy (PPMD) in Greensboro, North Carolina, the nonprofit announced. Determined to continue to make a difference in the life of Matthew Greiner and others who have Duchenne muscular dystrophy (DMD), the…

Long-term use of steroid treatment is linked to increased weight gain in boys with Duchenne muscular dystrophy (DMD) who are unable to walk, a study shows. Recognition of the effect of steroid therapy on patients’ weight and growth progression can have important implications on treatment management and early diagnosis of…