Muscular Dystrophy News

Muscular Dystrophy News We are dedicated to sharing the latest news, research, and muscular dystrophy patient perspectives.

Enrollment is now ongoing in a first-in-human clinical trial testing EPI-321, a therapy designed to target the underlyin...
08/08/2025

Enrollment is now ongoing in a first-in-human clinical trial testing EPI-321, a therapy designed to target the underlying cause of FSHD. https://buff.ly/h8iZWuv

Enrollment is now ongoing in a first-in-human clinical trial testing EPI-321, an epigenetic therapy designed to address ...
08/07/2025

Enrollment is now ongoing in a first-in-human clinical trial testing EPI-321, an epigenetic therapy designed to address FSHD. https://buff.ly/Jyqngz7

Living with Duchenne brings unique obstacles and challenges: https://bit.ly/46WhyX9This article covers key resources to ...
08/06/2025

Living with Duchenne brings unique obstacles and challenges: https://bit.ly/46WhyX9

This article covers key resources to help guide a daily care plan—from specialized therapy and assistive tools to local and online support communities, financial assistance, and advocacy pathways. With expert care and a strong support network, families can build independence and quality of life. 🧠

You don’t have to figure it all out alone. 💬 Whether you're newly diagnosed, a long-time caregiver, or somewhere in betw...
08/06/2025

You don’t have to figure it all out alone. 💬 Whether you're newly diagnosed, a long-time caregiver, or somewhere in between, our forums are a place to connect, ask questions, and share experiences with people who understand.

Real conversations. Real support. đź’šRight here, when you need it:
https://f.mtr.cool/zzwnintsgp

The FDA has granted breakthrough therapy designation to DYNE-251 for the treatment of DMD in patients amenable to exon 5...
08/06/2025

The FDA has granted breakthrough therapy designation to DYNE-251 for the treatment of DMD in patients amenable to exon 51 skipping. https://buff.ly/aBIWxmb

A strong care plan for ambulatory DMD isn’t just an option—it’s essential. https://bit.ly/41nQ1tLBy coordinating care am...
08/04/2025

A strong care plan for ambulatory DMD isn’t just an option—it’s essential. https://bit.ly/41nQ1tL

By coordinating care among neuromuscular specialists, PT/OT/speech therapists, nutritionists, and counselors, ambulatory individuals with DMD can maintain independence longer and enjoy a better quality of life.

They live it. They write it. 💬💪. https://f.mtr.cool/mdeukagykgMeet four incredible advocates and columnists who bring ho...
08/04/2025

They live it. They write it. 💬💪. https://f.mtr.cool/mdeukagykg

Meet four incredible advocates and columnists who bring honesty, heart, and firsthand experience to Muscular Dystrophy News. From caregiving insights to life with Duchenne and Becker, their stories offer support, perspective, and real-life connection to anyone navigating muscular dystrophy.

They're not just sharing—
they’re helping others feel seen, understood, and never alone. Get to know their voices:

Columnist Betty Vertin, whose days often feel overloaded, describes how it feels when her life as a caregiver is recogni...
08/01/2025

Columnist Betty Vertin, whose days often feel overloaded, describes how it feels when her life as a caregiver is recognized and affirmed. https://buff.ly/fVzd2vB

A mouse model study of Precision BioSciences’ gene-editing therapy PBGENE-DMD found it produced long-term functional imp...
07/31/2025

A mouse model study of Precision BioSciences’ gene-editing therapy PBGENE-DMD found it produced long-term functional improvement in muscles. https://buff.ly/UwXtBPZ

Sarepta will resume shipping Elevidys to ambulatory DMD patients in the U.S. after the FDA cleared the pause following i...
07/30/2025

Sarepta will resume shipping Elevidys to ambulatory DMD patients in the U.S. after the FDA cleared the pause following its review of safety data. The agency concluded that a recent death in Brazil was unrelated to treatment. https://bit.ly/3IMX1dz

This decision brings much-needed relief and critical therapy access to eligible Duchenne families—while highlighting the importance of careful monitoring moving forward.

When you’re living with Duchenne Muscular Dystrophy, taking care of your heart is just as important as managing muscle h...
07/30/2025

When you’re living with Duchenne Muscular Dystrophy, taking care of your heart is just as important as managing muscle health. https://bit.ly/3IVm91r

💚 This article offers simple, clear tips on how to support heart health—from checkups to daily habits—and how your care team can guide you along the way.

Columnist Robin Stemple treasured the time with his family during a recent weekend trip, but fears his FSHD will soon ma...
07/30/2025

Columnist Robin Stemple treasured the time with his family during a recent weekend trip, but fears his FSHD will soon make travel impossible. https://buff.ly/qGUXsEd

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