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What’s Ahead for Your 2026 National Ambassador
For over 70 years, MDA’s National Ambassadors have been an intrinsic part of championing for
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A caregiver’s birthday offers an opportunity to practice self-care
January doesn’t just bring a new calendar year. For four of us in this family,
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Solid Biosciences Shares Update on INSPIRE DUCHENNE Trial Evaluating SGT-003
Solid Biosciences, Inc., has shared updates on the company’s gene therapy program for the treatment
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Avidity Biosciences Managed Access Program (MAP) – Frequently Asked Questions
Avidity Biosciences has released answers to frequently asked questions about their Managed Access Program (MAP)
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New ‘pothole-filling’ RNA strategy targets the genetic root of DM1
Using a “pothole-filling” strategy that combines the precision of DNA-like strands with the versatility of
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Quest Podcast: Redesigning the Day: Accessibility and Mindset Life Hacks with Jax Cowles
In this Quest Podcast episode, we chat with public speaker, consultant, and disability advocate, Jax
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Solid Biosciences Reports Progress on Next-Generation Gene Therapy
Solid Biosciences reports that 33 children have now been dosed in their SGT-003 gene therapy
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What to Know Before You Start Adaptive Driving
For people living with neuromuscular diseases, driving can be more than a convenience — it
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FDA names once-daily (Z)-endoxifen an orphan drug for Duchenne
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to (Z)-endoxifen for
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Capricor provides regulatory update on Deramiocel for Duchenne
Capricor Therapeutics, which received early funding from CureDuchenne, said the FDA has requested the full
