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Awareness Initiatives and Innovative Therapeutic Approaches Bring Hope to People Living with Becker Muscular Dystrophy
The following content is sponsored by Edgewise Therapeutics and does not reflect the views of […]
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Dyne Therapeutics Shares New Clinical Data from DELIVER Trial of DYNE-251 in Duchenne Patients
Dyne Therapeutics has shared new clinical data from the ongoing DELIVER trial of DYNE-251 in […]
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Study to Explore Barriers to Diverse Clinical Trial Participation in Duchenne Published
PPMD is pleased to share that the article, “Barriers to diverse clinical trial participation in […]
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Dyne therapies showing benefit for MD patients, per clinical trial data
DYNE-101 and DYNE-251, Dyne Therapeutics’ investigational therapies for forms of muscular dystrophy, continue to work […]
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Dyne Therapeutics Announces New Clinical Data from ACHIEVE Trial of DYNE-101 in DM1 and DELIVER Trial of DYNE-251 in DMD Demonstrating Compelling Impact on Key Disease Biomarkers and Improvement in Multiple Functional Endpoints
As early investors in Dyne Therapeutics, we are pleased to share they reported positive updates […]
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PTC Therapeutics Announces European Commission Returns Translarna™ Opinion to CHMP For Re-evaluation
PTC Therapeutics, Inc. announced today that the European Commission (EC) has decided not to adopt […]
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Singing the ‘Bear Hunt’ song for the 30th time and loving it
Regular readers of my column will know that my wife, Wendy, and I moved from […]
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Duchenne Muscular Dystrophy Moves Closer to Newborn Screening Recommendation
**UPDATE** On May 10, 2024, the Advisory Committee on Heritable Disorders in Newborns and Children […]
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Lessons from final losmapimod trial data help inform future studies
Findings from a Phase 2 trial testing losmapimod in adults with facioscapulohumeral muscular dystrophy (FSHD) […]
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Congress Passes FAA Reauthorization Featuring Monumental Accessibility Improvements
This week marked a significant milestone as Congress successfully passed the bipartisan Federal Aviation Administration […]
