Duchenne’s Muscular Dystrophy (DMD) Resources

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There is a wealth of information available to people dealing with a DMD diagnosis. We have compiled a list of information for you on this page, including:

Resources you can access directly, including various local support groups
Videos with plenty of practical advice
Free downloads of DMD materials
Information on meetings and events about DMD

By providing you with these materials, we can help you better understand how to help manage DMD.

Downloadable materials and resources

A Summary of the DMD Care Considerations Working Group for Caregivers (English & Spanish) Learn more about care guidelines for DMD that address a multidisciplinary approach and the continuum of care for DMD management.

DMD comics for children

These printable comics have been created to help your child understand what DMD is and tell them about the team of people who will help them manage it. The comics include easy-to-understand adventures that can help your child navigate their DMD journey. These comics have been developed and funded by PTC Therapeutics.

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Coloring books

English

Spanish

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Caregiver considerations summary

English

Spanish

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Doctor’s appointment discussion guide

This quick-reference resource can help you prepare for your child’s next doctor visit.

Navigating scientific publications

This resource will help you to familiarize yourself with the elements and terms that are commonly seen in different types of scientific publications; very useful when reading medical journal articles.

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My Vibe

We believe every story in the rare disease community deserves to be told.
Insightful Moments My VIBE shares experience straight from the Voices of Inspirational, Brave, and Empowering patients and families. Click here to hear some of the most recent

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Duchenne guide for families

Developed by TREAT-NMD, this guide contains all the latest information on how DMD is diagnosed, how it progresses and the types of assistance available.

Take on Duchenne podcast

The Take on Duchenne North America podcast brings scientific leaders who work in the Duchenne Muscular Dystrophy (DMD) field to the table to share their knowledge. This podcast aims to educate and raise awareness of DMD among those interested in learning more about this progressive genetic disease.

Key DMD Scientific Publications

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A selection of key scientific papers and professional guidelines on the management of DMD. The ‘Navigating scientific publications’ resource to help you to familiarize yourself with the elements and terms that are commonly seen in different types of scientific publications.

How to reduce the time to a DMD diagnosis

Aartsma-Rus A, et al. J Pediatr. 2019;204:305–313.e4.
https://www.jpeds.com/article/S0022-3476(18)31550-6/fulltext

The importance of genetic testing for DMD

Aartsma-Rus A, et al. J Med Genet. 2016;53:145–151.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4789806/

International standard of care guidelines for the diagnosis and management of DMD

Part 1 Birnkrant DJ, et al. Lancet Neurol. 2018;17:251–267.
Part 2 Birnkrant DJ, et al. Lancet Neurol. 2018;17:347–361.
Part 3 Birnkrant DJ, et al. Lancet Neuroll. 2018;17:445–455.
http://www.treat-nmd.eu/care/dmd/diagnosis-management-DMD/

DMD care considerations: a guide for families

http://www.treat-nmd.eu/care/dmd/family-guide/translations/

The life expectancy of patients with DMD

Landfeldt E, et al. Eur J Epidemiol. 2020;35:643–653.
https://link.springer.com/article/10.1007/s10654-020-00613-8

The use of pre-natal screening to diagnose DMD before birth

Vita GL, Vita G. Neurol Sci. 2020;41:1677–1683.
https://www.ncbi.nlm.nih.gov/pubmed/32112218

Video Resources

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Max's story

Max Fernandez, a senior at Fordham University, has DMD. But his condition hasn’t stopped him from traveling the world and enjoying college life with the help of friends and family. In this video, Max shares his story, as well as challenges, common misconceptions about DMD, along with advice for kids and parents.

How to communicate with your healthcare providers

How to communicate with you healthcare providers transitioning from childhood to adulthood means moving from a pediatric medical practice to an adult practice—and this is especially important for people with DMD. Knowing what to say to a doctor, and how to say it, can be difficult, but this video has some useful tips. Sarah Tencer, MSW, talks about what to expect in your appointment, how to prepare for it, and what to do afterwards.

Challenging yourself

Challenging yourself can be scary—but there’s no better way to achieve and grow. In this video, Sarah Tencer, MSW, talks about how setting GROW and SMART goals can provide positive challenges to help you take your life in the direction that you want it to go. She also discusses resilience and identifying the tools that will help you to cope when things don’t go as planned.

Achieving independence

Achieving an independent lifestyle is important as people with DMD approach adulthood. In this video, Sarah Tencer, MSW, gives advice on managing money by setting realistic goals, and accessing public benefits. She also offers tips on entering employment, and things to consider when searching for a place to live.

Mechanism of disease

DMD resources en espanol

Managing transitions in DMD medical care

Amaris Sanchez, psychologist and expert in the formation of identity, talks about transitions in DMD medical care. This includes what needs to happen as part of an effective healthcare transition, who is involved and where to go for more information.

Guidelines for healthy eating

Eating well is essential to optimize the well-being and lifespan of people with DMD. In this video, dietitian nutritionist Sandra Arévalo-Valencia talks about how to select meals for healthy weight maintenance and bone health.

Physiotherapist-recommended stretches for DMD

Learn about the different stretches your physiotherapist may have recommended to be done at home to help prevent the loss of flexibility that comes with DMD.

Calf stretch

Hip flexors stretch

Knee and hamstring stretch

Wrist and hand stretch

Standing wedge stretch

IT band stretch

Wrist, elbow, and hand stretch

These videos are provided as examples only and are not intended to replace any advice given by your healthcare professional.

The stretches demonstrated in the videos should only be carried out if deemed suitable by your healthcare professional.

Audio Resources

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Below are a set of audio files recorded specifically for Spanish-speakers

What is DMD?

Early age DMD signs and symptoms – part 1

Early age DMD signs and symptoms – part 2

Seeking DMD diagnoses

Peer navigator Sima Perez

Genetic testing as diagnoses

Genetic family testing

Early diagnoses of DMD

Disease progression – part 1

Disease progression – part 2

Akari foundation

Treatment options

Advocacy Groups Are Ready to Help

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Advocacy groups play an important role in helping you connect and bond with other people affected by Duchenne as well as giving a public voice. The following patient advocacy groups are here to help by offering information, guidance, and support.

Advocacy groups in your community

Advocacy groups such as CureDuchenne, Muscular Dystrophy Association (MDA) and Parent Project Muscular Dystrophy (PPMD) are well known within the United States. Support is not limited to these groups and there are local community groups that can help you as well.

Cure Duchenne

Cure Duchenne partners with biotech and pharmaceutical companies, academic institutions, and other organizations to help find a cure for Duchenne muscular dystrophy.

https://cureduchenne.org/

Muscular Dystrophy Association (MDA)

MDA is a nonprofit health organization that sponsors research to find effective treatments for neuromuscular diseases.

https://mda.org/

Parent Project Muscular Dystrophy

PPMD is the largest, most comprehensive nonprofit organization in the United States focused on ending Duchenne muscular dystrophy through research, advocacy, care, and education.

https://www.parentprojectmd.org/

The Akari Foundation

The Akari Foundation is dedicated to supporting Hispanic immigrant and low-income families who live with Duchenne muscular dystrophy.

https://theakarifoundation.org

Jett Foundation

The Jett Foundation partners with individuals and families with Duchenne muscular dystrophy and other neuromuscular diseases. Offers empowering educational programming, direct service experiences, and by helps accelerate development of life-changing treatments.

https://www.jettfoundation.org

The Assistance Fund

For patients and families facing high medical out-of-pocket costs, the fund provides financial assistance to cover costs not paid for by insurance to patients with demonstrated need.

https://tafcares.org/

PTC Therapeutics does not endorse organizations that sponsor linked external websites, products, or services that such organizations may offer; and does not control or guarantee the currency, accuracy, relevance or completeness of the information found on the linked external sites.

All trademarks included herein are the property of their respective owners.

Find a certified Duchenne care center near you

Certified Care Centers provide comprehensive care and services to people living with Duchenne muscular dystrophy. The goal of these centers is to make comprehensive care available and accessible to as many patients with Duchenne and their families as possible. PTC does not warrant, whether expressed or implied, that the list of centers is accurate or complete.

The Muscular Dystrophy Association has a network of 200 specialized clinics across the United States and Puerto Rico, each with a team of healthcare professionals all at one location. Find a local clinic near you by visiting mda.org/locate, or by calling (800) 572-1717.

The Certified Duchenne Care Center Program was developed by Parent Project Muscular Dystrophy, with the help of many experts, to certify clinics and measure results, helping to ensure that centers maintain the highest standards in clinical care for Duchenne. Click here for more information and to locate a local certified center.

PTC does not recommend or endorse any center, hospital, or healthcare professional included above for any purpose, nor is the list to verify a center’s, hospitals, or specific healthcare professional’s credentials or qualifications. It is your responsibility to select a center, hospital, and/or healthcare professional appropriate to your needs, and you assume full responsibility for your interaction with any center, hospital, or healthcare professional you contact through the center list. PTC is not responsible for the medical care or advice given by any center, hospital, or healthcare professional you choose to contact.