Boys with Duchenne muscular dystrophy (DMD) may be able to regain lost height and overcome growth stunting by switching from traditional steroids to the newer medication Agamree (vamorolone), a new study suggests. While standard corticosteroids are essential for managing DMD-related inflammation, they are known to halt bone…
Duchenne muscular dystrophy
On Feb. 19, my girlfriend, Amanda, and I presented at a seminar titled “AI in Social Work” at the National University of Singapore’s School of Computing, on behalf of our accessible art studio, Rebirth Ensemble. It explored how we can harness artificial intelligence (AI) to drive social impact by…
Roche has decided to stop developing satralizumab for bone health in Duchenne muscular dystrophy (DMD), the company announced in a community letter. Patients already enrolled in the SHIELD DMD Phase 2 trial (NCT06450639) may continue on the study until the six-month bone mineral density collection, expected…
The U.S. Food and Drug Administration (FDA) has cleared Precision Biosciences to launch a clinical trial testing its gene-editing therapy candidate PBGENE-DMD in people with Duchenne muscular dystrophy (DMD) caused by certain mutations. Precision announced it had received a “Study May Proceed notification” from the U.S. regulatory agency…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to Dyscorban (ifetroban), Cumberland Pharmaceuticals’ treatment candidate for heart problems in Duchenne muscular dystrophy (DMD). Dyscorban has been tested in the Phase 2 FIGHT DMD trial (NCT03340675), with data showing that it improved heart…
For people with Duchenne muscular dystrophy (DMD), 2026 promises to be a year of tremendous excitement, with several new therapies poised for likely approval in the U.S., according to DMD advocates. “It’s an exciting time,” Debra Miller, founder CEO of CureDuchenne, said in an interview with Muscular Dystrophy…
In boys with Duchenne muscular dystrophy (DMD), executive skills — essentially the brain’s self-management tools, such as self-control, emotional regulation, and working memory — appear largely typical at age 5, begin to lag around age 8, and show signs of partial catch-up by early adolescence. Those are the findings…
New findings from research in mice are challenging longstanding beliefs about the causes of Duchenne muscular dystrophy (DMD), with evidence showing that the genetic disease is marked by abnormalities in muscle stem cells during fetal development — indicating DMD may start in the womb. According to the researchers, these findings suggest…
Turning 30 last month was a significant milestone in my Duchenne muscular dystrophy (DMD) journey. For years, my care has revolved around keeping my vital functions strong enough to sustain a body whose muscles continue to wither with every passing second. Having lived with DMD since birth, I’ve witnessed…
A couple weeks ago, I co-emceed the Zero Project Asia Pacific Symposium 2025 with my girlfriend, Amanda. The event, a collaboration between SG Enable and the Zero Project, took place at Enabling Village, a community space here in Singapore designed for accessibility. The symposium gathered leaders in Asia to share ideas…
Recent Posts
- To me, a wheelchair is a symbol of improved function, not disability
- When school is out, my sons with DMD get into their summer groove
- Upsher-Smith launches new DMD support websites for Kymbee treatment
- Exploring other mobility devices when a walker is no longer sufficient
- Collaboration aims to improve design of FSHD clinical trials