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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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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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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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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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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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Congress Passes FAA Reauthorization Featuring Monumental Accessibility Improvements
This week marked a significant milestone as Congress successfully passed the bipartisan Federal Aviation Administration […]
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Del-desiran for DM1 wins FDA’s breakthrough therapy designation
The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to delpacibart etedesiran […]
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Mapping Out a Path Forward: PPMD Convenes Representatives from 21 Academic Centers and 11 Industry Partners for 2024 Cardiac Care Workshop
Last week, PPMD held the 2024 Cardiac Care Workshop in Baltimore, Maryland, which included 40 […]
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Meeting the challenges of DMD with laughter, love, and learning
Life with Duchenne muscular dystrophy (DMD) doesn’t get easier as time goes by, but it’s […]
