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Sevasemten lowers markers of muscle damage in BMD: Trial
The investigational oral therapy sevasemten significantly reduced markers of muscle damage in people with Becker muscular dystrophy (BMD) treated in a Phase 2 trial, according to top-line results announced by the therapy’s developer, Edgewise Therapeutics. “This landmark study presents compelling biomarker data and promising signals that suggest the potential for functional stabilization with administration of…
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New drug shows promise against Duchenne muscular dystrophy
A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration. Researchers have discovered that an experimental compound called K884 can boost the natural repair abilities of muscle stem cells. Current treatments can slow muscle damage, but don’t address the root problem.
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How my teaching job reminds me that I’m more than a caregiver
I’m more than a caregiver, but it took a recent life change for me to realize that. I’m a mom to seven children I share with my husband, Jason, including three of our sons — Max, 19, Rowen, 15, and Charlie, 13 — who live with Duchenne muscular dystrophy (DMD). Their DMD diagnoses happened in…
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Agamree recommended for NHS use in UK, approved in China
Agamree (vamorolone) has been recommended for use in the National Health Service (NHS) in England, Wales, and Northern Ireland for treating Duchenne muscular dystrophy (DMD) patients, 4 and older. The recommendation came from the U.K.’s National Institute for Health and Care Excellence (NICE) and follows Agamree’s approval in the U.K. early this year. Agamree, a…
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Advance the Research Evolution: Introducing PPMD’s Dystrophinopathy Clinical Research Network
In 1984, the word “Duchenne” entered my world. The progress we have made since then is astounding. For decades, we have united in our unwavering commitment to end Duchenne, and with recent achievements in this fight, we are experiencing a new reality where more individuals living with Duchenne and Becker have access to therapies than…
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I’ll continue to do what I love, despite the FSHD ‘hangovers’
My wife, Wendy, and I had a wonderful Sunday recently. We rose early and hit the road to Shanksville, Pennsylvania, where we had lived for over 41 years before moving to Pittsburgh to be closer to our kids and grandkids. I’d agreed to play for the 10:30 a.m. service at our former church, the Unity…
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$10M in prizes offered toward work on FSHD treatments
The venture philanthropy organization SOLVE FSHD is offering $10 million in prizes for innovators who are working to develop new treatments for facioscapulohumeral muscular dystrophy (FSHD). “This competition will bring together the brightest minds in medicine, technology, and science, all working toward a cure for this devastating disease,” Eva Chin, executive director of SOLVE FSHD,…
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Catch up on PPMD Together: Virtual Edition – A Gathering of Families for Connection, Support, and Growth
Last month, families from around the world came together for PPMD Together: Virtual Edition. This unique virtual gathering provided families a space to connect, share their experiences, and find support from one another as they navigate the daily challenges of living with Duchenne and Becker muscular dystrophy. Following on the heels of two successful in-person…
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Arrakis’ RNA-targeted oral therapies showing promise for DM1
Arrakis Therapeutics’ investigational RNA-targeted small molecule (rSM) therapies were found to work as intended in preclinical studies and eased symptoms in a mouse model of myotonic dystrophy type 1 (DM1). The company presented the findings at the Cell Symposia: Chemical Biology in Drugging the Undrugged conference, held earlier this week in San Francisco. “We are…
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When art and advocacy meet onstage
Last Saturday, I performed a monologue titled “Connection” at the Enabling Lives Festival here in Singapore. ART:DIS, a local nonprofit that uses the arts to support people with disabilities, organized the event as part of its BEYOND DIS:PLAY performing arts and theater mentorship program. The festival centered on a development showcase, titled “The Human Condition.”…
