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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 […]
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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 […]
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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 […]
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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, […]
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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
