According to an article from Marisa Wexler in Muscular Dystrophy News, promising study data is available from the Phase 1 ARCH study. Within the study, researchers evaluated EDG-5506, an…
Funding for clinical trials can be an issue, especially for rare disease studies. Luckily, there are programs that exist to help with the financials, such as the "Clinical Studies of…
For years, researchers have evaluated Italfarmaco's givinostat for patients with Duchenne muscular dystrophy (DMD). In fact, a Phase 2/3 clinical trial showed that long-term givinostat treatment helped slow respiratory decline…
Emflaza (deflazacort), a corticosteroid, was originally approved by the FDA in 2017 to treat Duchenne muscular dystrophy (DMD) in patients five years or older. This approval was expanded in 2019…
Edgewise Therapeutics has announced their plan to initiate a Phase 1 trial of EDG-5506, their small molecule therapy for Becker muscular dystrophy. It will take place at Worldwide Clinical Trials…
With 4.15 million cases across the globe, COVID-19 is at the forefront of discussion. Knowledge about the viral pandemic continues to grow each and every day. Yet there are still…
The Cure Rare Disease Foundation The Cure Rare Disease Foundation's mission is to increase collaborative efforts between researchers in order to accelerate the development of customized therapies for individuals living with…
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