I’m now the one giving care since my caregiver’s illness

Note: This column contains spoilers for the series finale of the Netflix TV show “Dead to Me.” As I write this, the date is April 3. I’ve just survived the longest and most helpless week of my life. Two Sundays ago, my support worker, Glenda, suddenly fell ill from…

A rare neurological problem that does not appear to be directly related to AOC 1001 — the experimental treatment for myotonic dystrophy type 1 (DM1) being given patients in a Phase 1/2 clinical trial — may be the reason for a partial hold placed on the study,…

As a child, I wasn’t around people who used power wheelchairs. Perhaps because my sons were the first people I’d met who were living with Duchenne muscular dystrophy (DMD), or maybe because I’m old enough, power chairs seemed less prevalent when I was younger. After my sons were…

DYNE-251, an investigational therapy for Duchenne muscular dystrophy (DMD), has received orphan drug and rare pediatric disease designations from the U.S. Food and Drug Administration (FDA). Orphan drug status is meant to support the development of therapies for rare conditions affecting less than 200,000 people in the U.S. It…

I was raised to put my best foot forward, keep a stiff upper lip, and always keep going. My favorite Bible verse is Romans 8:28: “And we know that all things work together for good to those who love God, to those who are the called according to…

Treatment with Translarna (ataluren) helps to slow the decline in motor function for people with Duchenne muscular dystrophy (DMD) caused by nonsense mutations, according to new clinical trial data and analyses presented this month at the Muscular Dystrophy Association’s MDA Clinical & Scientific Conference. The work was funded…

Note: This story was updated March 31, 2023, to clarify that the presentation was given at the Muscular Dystrophy Association’s MDA Clinical & Scientific Conference. Three boys with Duchenne muscular dystrophy (DMD) treated with the experimental cell therapy DT-DEC01 in a clinical trial continue to show improvements in motor…

As a mom and caregiver to three sons with Duchenne muscular dystrophy (DMD), my focus is always on someone else, and I tend to slip lower and lower on the priority list. I’ve been thinking about this a lot lately. Since my oldest son, Max, 17, broke…

Four boys with Duchenne muscular dystrophy (DMD) who were treated with the gene therapy candidate SRP-9001 (delandistrogene moxeparvovec) in a proof-of-concept clinical trial continue to show improvements in motor function four years after dosing. That’s according to new data discussed this month at the Muscular Dystrophy Association’s MDA Clinical…

On the evening of March 8, I was mindlessly scrolling through my Facebook feed when I stumbled upon a public post from Singaporean Paralympic swimmer Sophie Soon, who uses a guide dog for help with her visual impairment. In the post, which has now been deleted, Soon expressed grievances with…