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There’s never a dull moment living with Duchenne muscular dystrophy
Living with Duchenne muscular dystrophy (DMD) has involved a series of challenges and triumphs, each […]
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PepGen Announces Clinical Hold in the U.S. on IND Application to Initiate CONNECT2-EDO51 Phase 2 Study of PGN-EDO51
PPMD is disappointed to learn that PepGen Inc. has received a full clinical hold notice […]
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PepGen’s DMD Exon 51 Skipping Program On Clinical Hold In US
PepGen has received a clinical hold notice from the FDA regarding its Investigational New Drug […]
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Edgewise Therapeutics Announces Positive Topline Results from CANYON Phase 2 Trial of Sevasemten in Individuals with Becker
Edgewise Therapeutics, Inc., has announced positive topline results from the double-blind, randomized, placebo-controlled Phase 2 […]
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Edgewise announces Topline results for Becker Phase 2 Trial
As an early funder of Edgewise, CureDuchenne is pleased to share that the Phase 2 […]
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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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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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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, […]
