My oldest son with Duchenne muscular dystrophy, Max, starts his sophomore year in high school this week. He will be halfway through his high school education by the end of the school year. For a long time, I was afraid to look too far into his future. At diagnosis, a…
The U.S. Social Security Administration (SSA) has added Duchenne muscular dystrophy (DMD) to its Compassionate Allowances (CAL) program list, accelerating the disabilities application process for adults with this genetic disorder. The program is designed to identify severe medical diseases and conditions that automatically meet the SSA’s standards for…
I wanted to expand on my last column by delving into the types of questions we should ask when presented with a new ailment, such as muscular dystrophy. I’d like to start with an anonymous quote: “It ain’t what people don’t know that hurts them, it’s what they know…
The U.S. Food and Drug Administration (FDA) has granted fast track status to EDG-5506, Edgewise Therapeutics’ investigational oral therapy for Becker muscular dystrophy (BMD). This designation is given to experimental treatments that are intended to treat serious conditions and fill an unmet medical need. It confers certain benefits, like…
I came down with a nasty cold two weeks ago, and even though I was sick, felt terrible, and knew I needed to rest as much as possible because I also have Duchenne muscular dystrophy, I was still judging myself for not working toward my looming deadlines. I’ve…
I’m a glass-half-full kind of girl. It’s usually not difficult for me to look on the bright side of life. Recently, I had one of those days where everything felt right. We are on top of my 2-year-old son Alfie’s therapies, he is loving day care, the equipment he…
My kids return to school next week. As always, there is so much to do to get ready. In addition to back-to-school shopping and open house, I have to ensure Max, Rowen, and Charlie, my sons with Duchenne muscular dystrophy (DMD), will be successful and that their school is…
PepGen has raised funding totaling $112.5 million to advance toward the clinic its enhanced delivery oligonucleotide (EDO) lead programs in Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1). The funding round keeps PepGen on track to start Phase 1 clinical trials in 2022 for DMD, and…
Losmapimod, a potential oral treatment for facioscapulohumeral muscular dystrophy (FSHD), missed its main goal in a recent Phase 2 clinical trial, but its developer, Fulcrum Therapeutics, sees this as a constructive failure. Lessons learned will help to shape losmapimod’s continued development, which is supported by the secondary…
A hostess I am not! Planning a child’s birthday party or hosting a team meal is the extent of my party planning skills. I am usually introverted and quiet, and only a few people will hear me talk much. Writing is my favorite way of communicating, and it has led…
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