Most 3-year-olds only get to watch marathons from the sidelines or on television, but not Levi Gagner. The Rare Disease Report revealed that Levi, along with his dad Kyle, completed the Rock ‘n’ Roll half marathon earlier this year. The Pheonix, Arizona event is the third race the two have competed…
Respiratory activity is significantly impaired in Duchenne muscular dystrophy (DMD), but a new mouse study suggests that enhancing breathing via the brain may improve respiratory capacity. The study “Sensorimotor control of breathing in the mdx mouse model of Duchenne muscular dystrophy” was published in The Journal of Physiology. Respiratory failure…
As boys with Duchenne muscular dystrophy get older, their disease progresses and it’s likely that their emotional response to the disease will change over time. It’s perfectly natural for Duchenne MD patients to experience frustration, anger, self-pity and anxiety regarding their condition, but there are ways that you can help…
Researchers have found that reducing levels of elevated sarcolipin protein may be a potentially effective therapeutic strategy in the treatment of Duchenne muscular dystrophy (DMD) patients. The study, “Reducing sarcolipin expression mitigates Duchenne muscular dystrophy and associated cardiomyopathy in mice,” was published in Nature Communications. Current therapies…
Crutches have long been a cumbersome device that designers haven’t thought twice about — until now. As you can see in this video from max, crutches have finally been given a much-needed makeover. MORE: Wearable robot hand could give muscular dystrophy patients better hand control Instead…
More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in clinical studies that will test new ways of evaluating — and advancing — potential treatments, including the use of natural history…
Casey’s General Stores customers contributed $1.35 million in August to support the Muscular Dystrophy Association‘s 2017 Summer Camp, where children living with MD enjoy a week of fun and make new friends at no cost to their families. Since the fundraising campaign was launched in 2006, $9 million has been…
Two non-profits that work on standards that can facilitate drug development have created a guide to help those doing Duchenne’s muscular dystrophy research. The two are Critical Path Institute (C-Path) and the Clinical Data Interchange Standards Consortium (CDISC). The resource they created is the Duchenne Muscular Dystrophy Therapeutic Area User…
As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies to develop therapies for rare diseases — off the table. That’s the message being pushed by the National Organization for…
In this animated video from the Parent Project Muscular Dystrophy, Tim and Moby talk about Duchenne muscular dystrophy in a way that children of all ages can understand. MORE: Five tips for emotionally coping with Duchenne muscular dystrophy The cartoon duo explains that Duchenne muscular dystrophy (DMD) affects approximately…
Get regular updates to your inbox.