Muscular Dystrophy Pakistan

01/08/2026

New analyses from the Phase 3 EPIDYS clinical trial are providing additional insight into Duvyzat’s potential benefits for boys with Duchenne muscular dystrophy (DMD). Read more here: https://bit.ly/4eQq8dN

According to researchers, patients receiving the therapy experienced functional gains regardless of the dose level they ultimately received. MRI findings also suggested slower muscle deterioration, including reduced fat infiltration and slower loss of functional muscle tissue.

01/08/2026

For people living with muscular dystrophy, pressure sores can become a major issue—but they’re not inevitable. https://bit.ly/4oiuwnO

According to Muscular Dystrophy News, regular skin monitoring, pressure relief, and skin-friendly care routines can significantly reduce the risk. 🔍

28/07/2026

16/07/2026

Avidity Biosciences, Inc. announced the submission of a New Drug Application to the U.S. Food and Drug Administration (FDA) for delpacibart zotadirsen. Also referred to as "del-zota" or AOC 1044, the investigational therapy is being proposed for the treatment of individuals with Duchenne muscular dystrophy (DMD) who possess a genetic mutation amenable to exon 44 skipping (DMD44).

Please read the letter full.

15/07/2026

Support LGMD Awareness Day 2026

On 30 September 2026, Muscular Dystrophy Pakistan will observe Limb-Girdle Muscular Dystrophy (LGMD) Awareness Day to raise awareness, educate communities, support affected individuals and families, and promote early diagnosis and access to care.

We invite individuals, companies, and organizations to support this important initiative through donations or sponsorships. Your contribution will help us organize awareness campaigns, educational materials, patient support programs, and community outreach activities.

Together, we can create hope, spread awareness, and improve the lives of people living with Limb-Girdle Muscular Dystrophy (LGMD).

To donate or become a sponsor, please contact Muscular Dystrophy Pakistan.

14/07/2026

Support DMD & BMD Awareness Day 2026

On 7 September 2026, Muscular Dystrophy Pakistan will observe Duchenne (DMD) & Becker (BMD) Muscular Dystrophy Awareness Day to educate communities, support affected families, and promote early diagnosis.

We invite individuals, companies, and organizations to support this important initiative through donations or sponsorships. Your contribution will help us organize awareness activities, educational materials, and patient support programs.

Together, we can create hope, spread awareness, and improve the lives of people living with muscular dystrophy.

To donate or become a sponsor, please contact Muscular Dystrophy Pakistan.

09/07/2026

The Speak Foundation's 2026 LGMD Scientific Summit on July 31st at 11:00AM EDT will bring together leading researchers, expert clinicians, and innovative biotech companies to share the latest advances in limb-girdle muscular dystrophy research and care. Learn about emerging therapies, clinical trial updates, and the future of LGMD treatments directly from the experts driving progress.

Whether you are living with LGMD (or are suspected to), caring for someone who is, working in the medical field, or passionate about advocacy and research, this FREE event is designed to inform, equip, and empower you.

The full Summit schedule is published in our Summer issue of LGMD News magazine which just arrived in US mailboxes, will be sent out digitally to those who are a part of our patient network, and can be found on our website www.thespeakfoundation.com

Take a look at this incredible lineup of speakers below!

We would like to extend a special Thank You to our Platinum sponsor, BridgeBio, and our Bronze sponsor, AskBio.

Register for the summit today using this link: https://tinyurl.com/2026LGMDSummit

06/07/2026

The Stiff Elbows That Hide a Deadly Heart Risk | EDMD | MDP

25/06/2026

🎉 We're Live! 🌐

Muscular Dystrophy Pakistan is proud to announce the launch of our official website.

Our website has been developed to serve as a dedicated platform for raising awareness about Muscular Dystrophy and Rare Diseases, sharing educational resources, highlighting our programs and initiatives, and connecting patients, families, healthcare professionals, researchers, and advocates across Pakistan and beyond.

Through this platform, visitors can learn more about our mission, ongoing projects, upcoming events, advocacy efforts, and opportunities to support the rare disease community.

We invite you to explore our website and join us in our commitment to improving the lives of individuals and families affected by Muscular Dystrophy and other Rare Diseases.

🌐 Visit our website: https://musculardystrophypk.org

Together, we can build a more informed, inclusive, and supportive future for the rare disease community.



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