Astellas Gene Therapies has terminated research and development of its gene therapy programs AT702, AT751, and AT753 for Duchenne muscular dystrophy (DMD). The move was based on recent preclinical data, the company announced in a press release. AT702, AT751, and AT753 are exon skipping medicines designed…
When I went off to school at the University of Southern California (USC), it was the first time, like many other 18-year-olds, that I would be living away from home. It was an especially huge change for me because of my Duchenne muscular dystrophy and my reliance on my…
I’m late to pick up the kids from school a lot now. I haven’t always been this way, though. Last year, I’d arrive 10 minutes early every day and use it for some quiet prayer time. But since having a baby earlier this year, I arrive late to school…
A new natural history study that seeks to collect information about how Becker muscular dystrophy (BMD) progresses in the absence of treatment is now enrolling. The study is being led by the GRASP (General Resolution and Assessments Solving Phenotypes) consortium and Virginia Commonwealth University, its primary sponsor. Also collaborating…
Twins Oscar and Sebastian Spink, 11, may not be your typical “Avengers” superheroes, but they are certainly pursuing a gargantuan task that would have impressed even Tony Stark. The boys, diagnosed with facioscapulohumeral muscular dystrophy (FSHD) in 2019 and die-hard fans of the Marvel Cinematic Universe, are attempting to…
The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…
We throw around the word “community” a lot in the rare disease space, but don’t always think about what it really means and how it’s vital for patients, family, friends, and caregivers. I rarely see anyone in a wheelchair in public, let alone a power chair. Growing up, I didn’t…
My family has been on the Duchenne muscular dystrophy (DMD) journey for almost 12 years. During that time, I’ve quietly watched how friendships and making friends have changed for each of my three sons with DMD as they have grown. I’ve traveled farthest down this road with my oldest…
DT-DEC01, Dystrogen Therapeutics’ experimental chimeric cell therapy for Duchenne muscular dystrophy (DMD), appears generally safe and improved muscle strength and motor function in the first three boys enrolled in a Phase 1 clinical trial. Based on these positive results of the therapy’s low dose, with follow-up ranging from one…
The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues in a nod to its increased use during the ongoing COVID-19 pandemic. NORD’s report card project began seven…
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