News

With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Credit and affordable health insurance to future funding for rare disease research. Yet “whether Democrats take over the House or Senate,…

Muscle biopsies can benefit the diagnosis and treatment of neuromuscular diseases in children, particularly those with muscular dystrophy or inflammatory muscle disease, a study suggests. In the study, “Does muscle biopsy change the treatment of…

To raise money for treatments and to support families touched by muscular dystrophy, the Muscular Dystrophy Association’s (MDA) New York Muscle Team will present its 22nd annual gala Dec. 3 in New York City. To be held at Cipriana Wall Street, the event will feature former New York…

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…