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Specialty pharmacies: a guide for Duchenne muscular dystrophy patients & carers

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Specialty pharmacies: a guide for Duchenne muscular dystrophy patients & carers

Exciting breakthroughs in rare disease treatments mean more patients are gaining access to cutting-edge therapies - and specialty pharmacies are playing a crucial role in delivering these medications with expert support. But what exactly do they do, and when do they come into play?

AllMyHealth’s latest guide breaks it all down - exploring how specialty pharmacies work, their expanding role in patient care, and how individuals can engage with them. Plus, we spotlight specialty pharmacies such as Orsini and Accredo which provide DMD treatment.

Available in both text and audio formats - dive in today and share with your community!

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The report is available for free online on the AllMyHealth website.

www.allmyhealth.io/reports

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Latest Research

In their review, Zhao et al. (2025) discuss how the fruit fly, Drosophila melanogaster, is an effective model for studying muscular dystrophy. They highlight the benefits of using this organism, such as its genetic similarity to humans and the ease of manipulating its genes. This has allowed researchers to better understand the disease's progression, the function of specific genes related to muscle health, and to identify potential new targets for treatment.

The review emphasizes the insights gained from Drosophila models, which have been instrumental in unraveling the complex molecular mechanisms of various types of muscular dystrophy. By studying these flies, scientists have been able to explore the genetic underpinnings of muscle weakness and degeneration, which is crucial for developing future therapies.

Community News

Muscular Dystrophy AssociationMuscular Dystrophy AssociationFeb 06, 2025

🚨 Medicaid is at risk - we need your voices now!

Millions of Americans, including the neuromuscular community, rely on Medicaid for access to health care. Proposed Congressional changes - funding cuts, work requirements, & more - put care at risk.

Take action: MDA.org/Medicaid

Muscular Dystrophy Association Post
Muscular Dystrophy UKMuscular Dystrophy UKFeb 04, 2025

Yvonne has been a single mum for most of her children’s lives, raising two boys with Duchenne muscular dystrophy. 🧡

She lost her eldest son, Casey at her local hospice. Her youngest son, Shane, now 42, has been told he's one of the oldest men with Duchenne in the country.

“Shane has always been independent—he has his own flat and has even had girlfriends,” Yvonne shares.

Through it all, Yvonne has navigated this journey largely on her own after her husband struggled to cope with the boys' diagnosis.

“For most of my children’s lives, I had nobody to support me... I had to figure things out on my own.”

Finding one of our local support groups was a turning point for Yvonne. "It gave me comfort, made me feel less alone, and we even went on days out together.” 🤝

Her advice to other parents? "Make lasting memories. Let them do whatever they can— even if you’re unsure, let them try."

Read their full story here: https://loom.ly/o244fAU

#Duchenne

Muscular Dystrophy UK Post
Harrison & DuchenneHarrison & DuchenneFeb 06, 2025

#duchennemusculardystrophy#resilience

Harrison & Duchenne Post

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