Amid a topsy-turvy start to 2024, I’ve marked a date on my calendar I’ve been looking forward to more than any other: Valentine’s Day. While it might not be a particularly significant day for many others, it’s a big deal for me. It’ll be my first time celebrating the day…
It may take an average of 11 years before people with Duchenne muscular dystrophy (DMD) lose upper body function and the ability to breathe on their own, according to a study by researchers of the HERCULES project, which set out to construct a natural history model of the…
I do too much. You might assume I’m talking about caregiving because you’re reading a column written by a caregiver. And in part, you’re right. But mostly, it’s an admission that I’ve been doing things for my three sons with Duchenne muscular dystrophy (DMD) that they can…
A committee of the European Medicines Agency (EMA) has maintained its initial recommendation not to renew the conditional marketing authorization for Translarna (ataluren), PTC Therapeutics’ medication for Duchenne muscular dystrophy (DMD). The recommendation by the Committee for Medicinal Products for Human Use (CHMP) comes after the company appealed…
I received an email on Jan. 12 from Duchenne UK, an important Duchenne muscular dystrophy (DMD) nonprofit in the United Kingdom. Its announcement: “Breaking News! First treatment for all [DMD] patients approved in U.K.” It went on to note that the Medicines and Healthcare products Regulatory Agency,…
SRP-5051 is able to increase the production of dystrophin protein in people with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping — a trial finding its developers suggest will lead to a positive risk-benefit profile for the next-generation DMD treatment. The full results of the two-part…
I love to read, but finding time to sit down with a good book can be challenging in my busy house. As a mom to seven and a primary caregiver to my three sons with Duchenne muscular dystrophy (DMD), quiet time is often interrupted by my children. Occasionally, however,…
The U.S. Food and Drug Administration (FDA) has given orphan drug designation to JUV-161, Juvena Therapeutics’ lead treatment candidate for myotonic dystrophy type 1 (DM1). Orphan drug status is designed to encourage the development of therapies for rare diseases, or those affecting fewer than 200,000 people in the U.S. It provides…
The symptoms of my facioscapulohumeral muscular dystrophy (FSHD) make staying positive a daily challenge. Many people with muscular dystrophy struggle with depression. This battle is magnified every winter as I also feel the impact of seasonal affective disorder (SAD). Exposure to sunlight helps our bodies…
Sarepta Therapeutics has started screening participants for a Phase 3 clinical trial called EMERGENE that will test its gene therapy candidate SRP-9003 in children with limb-girdle muscular dystrophy type 2E (LGMD2E). The open-label trial, also known as SRP-9003-301, aims to recruit 15 patients, age 4 and older, with or without…
Get regular updates to your inbox.