Why lending a hand to a person with FSHD should make the news

Why lending a hand to a person with FSHD should make the news

Getting surprise help when you have facioscapulohumeral muscular dystrophy (FSHD) isn’t considered newsworthy. But maybe it should be. These days, it seems that most, if not all, of the news on television and social media is bad. These negatives aren’t limited to reports on wars, hurricanes, inflation, pandemics, and…

Oral AMO-02 aids muscle, heart health in mouse model of DMD

AMO-02 (tideglusib), an experimental oral therapy for certain muscular dystrophies, showed promise in boosting motor and cardiac muscle health in a mouse model of Duchenne muscular dystrophy (DMD). According to AMO Pharma, the treatment’s developer, AMO-02 also improved the animals’ metabolism and cognitive abilities. “These studies…

How Duchenne deters dreams about the future

I drove my oldest son, Max, who is 17, to his second college visit this week. As we veered onto the interstate, I told him I knew someone his age who’d already decided to attend the school we were about to visit. He was surprised. “Already?” he said. “How…

CureDuchenne to offer free new virtual resource for DMD caregivers

Covering topics such as mobility equipment and the role of physical therapy, a new, free, online CureDuchenne course seeks to provide guidance and support to Duchenne muscular dystrophy (DMD) caregivers. Specifically, the virtual CureDuchenne Caregiver Course seeks to help caregivers of those newly diagnosed with DMD —…

Translarna approval for DMD in EU not recommended for renewal

The conditional approval of Translarna (ataluren) in Europe as a treatment for Duchenne muscular dystrophy (DMD) — in place for nearly a decade — should now not be renewed, a committee of the European Medicines Agency (EMA) has recommended. The EMA’s Committee for Medicinal Products for Human Use…

Making modifications to curb the exhaustion of high school

Duchenne muscular dystrophy (DMD) is not a one-size-fits-all disease. I have three sons with DMD: Max, 17, Rowen, 14, and Charlie, 12. Each differs vastly from the others, from the speed of disease progression to how their bodies handle the changes, plus how each deals with the stress and…

Enrollment complete in Phase 3 trial of losmapimod for FSHD

Fulcrum Therapeutics has completed patient enrollment for its Phase 3 clinical trial evaluating losmapimod as a potential treatment for people with Facioscapulohumeral muscular dystrophy (FSHD). The study, dubbed REACH (NCT05397470), is evaluating the efficacy and safety of losmapimod against a placebo in FSHD adult patients across…

When did standing up become a multistep process?

Back in the 1980s, as a high school special education teacher, I enjoyed giving my students an assignment at the start of the school year to see if they could follow directions. I told them to read the document I handed out before doing anything. The directions on the top…

The DMD community reminds me of my purpose in life

On Sept. 2, the Muscular Dystrophy Association (Singapore) (MDAS) held its annual Go the Dystance carnival at One Punggol, a community hub that houses the Punggol Regional Library. The event is typically held between SMA Awareness Month in August and World Duchenne Awareness Day on Sept. 7.

$25K grants will help nonprofits counter DMD healthcare disparities

Entrada Therapeutics has awarded $25,000 grants to three U.S. nonprofits to aid in their efforts to combat healthcare disparities in the Duchenne muscular dystrophy (DMD) community. The goal of these nonprofits, as inaugural recipients of the biopharmaceutical company’s DREAMS — Diversity, Representation, Equity and Advocacy Matters — grant…