Sarepta Therapeutics is acquiring from Arrowhead Pharmaceuticals the exclusive global rights to develop ARO-DUX4 and ARO-DM1, two RNA interference (RNAi) therapeutic candidates in Phase 1/2 clinical testing, each for one type of muscular dystrophy. ARO-DUX4 is being tested for facioscapulohumeral muscular dystrophy (FSHD) and ARO-DM1 for…
I think about things deeply, rolling ideas around in my head until they make sense. My creative side, the writer, likes to wonder in this way. And as an introvert who naturally tends to keep everything to myself, writing is often the way I can express ideas and feelings after…
A woman who developed heart failure due to Duchenne muscular dystrophy (DMD) was diagnosed and treated before she got pregnant, which led to a healthy outcome for her and her baby girl, according to researchers in Japan. The woman’s case was reported in “Successful Pregnancy Outcome With Preconception…
I’m getting more comfortable using my walker. However, it just doesn’t pay to trust the darned thing. The other day in my bedroom, I was putting clothes away. I was planning to take a shower later in the day, so I thought I’d save myself some steps. I grabbed…
GNT0004, an experimental gene therapy for Duchenne muscular dystrophy (DMD), appears to be working as intended in the initial parts of a multiphase clinical trial, with benefits including stable or improved motor function. That’s according to data presented by Genethon, the therapy’s developer, at the ASGCT Breakthroughs in…
The power to influence or even direct people’s behavior or the course of events is part of a typical definition of “control.” When living with a chronic illness, as I am with limb-girdle muscular dystrophy, our desire to control the course of events can be a creative balancing act.
Life feels busy, which is no surprise to me. My husband, Jason, and I share seven children: Lexi, 23; Max, 19; Chance, 17; Rowen, 15; Charlie, 13; Mary, 10; and Callie, 2. Max, Rowen, and Charlie live with Duchenne muscular dystrophy (DMD). Our lives are busy year-round. This year…
Regenxbio has initiated the pivotal phase of a clinical trial testing its experimental gene therapy RGX-202 in boys with Duchenne muscular dystrophy (DMD). If the results are positive, they could support the therapy’s accelerated approval by the U.S. Food and Drug Administration (FDA). Regenxbio has also announced…
On Saturday, I presented at the Participation — Inclusion in Action Conference 2024, which was held in my home of Singapore for the first time. My physiotherapist at the National University Hospital, who’s also a disability and healthcare researcher and one of the event’s key organizers, invited me to…
A new technology called StitchR effectively restored the production of large muscle proteins dystrophin and dysferlin in mouse models of Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophy (LGMD), according to a study. The results have potential implications for gene therapies. The technology works by delivering two halves…
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