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℅ Raymond A. Huml, Quintiles Inc., 4820 Emperor Blvd, Durham, NC 27703, USA
For newly diagnosed muscular dystrophy (MD) patients and their caregivers in the U.S., the first point of call for information and support should be the Muscular Dystrophy Association (MDA, www.mda.org). In addition, there are disease-specific groups for several of the nine forms of MD, as shown in Table 13.1.
Table 13.1
Patient advocacy groups for MD
Type of MD | Sources of information and support |
|---|---|
Duchenne MD | MDA (MDA, mda.org) |
Parent Project Muscular Dystrophy (PPMD, http://www.parentprojectmd.org) | |
The Foundation to Eradicate Duchenne (http://duchennemd.org) | |
Duchenne Alliance (http://www.duchennealliance.org) | |
Other organizations around the world are listed at http://www.parentprojectmd.org/site/PageServer?pagename=Connect_partners, http://www.treat-nmd.eu/dmd/patient-organizations/, https://www.duchenneconnect.org, & http://www.cureduchenne.org | |
Becker MD | |
Congenital MD | MDA (MDA, mda.org) |
Cure CMD (http://curecmd.org) | |
Distal MD | |
Emery-Dreifuss MD | |
National Organization for Rare Disorders (NORD, https://www.rarediseases.org) | |
Facioscapulohumeral MD (FSHD) | FSH Society (http://www.fshsociety.org) |
MDA (MDA, mda.org) | |
PNW Friends of FSH Research (http://www.fshfriends.org) | |
Limb-girdle MD | MDA (MDA, mda.org) |
Jain Foundation (http://www.jain-foundation.org) | |
Myotonic dystrophy (DM) | MDA (MDA, mda.org) |
Myotonic Dystrophy Foundation (http://www.myotonic.org) | |
Oculopharyngeal MD (OPMD) | |
All Forms of MD
The MDA is the world’s leading nonprofit health agency dedicated to finding treatments and cures for MD, amyotrophic lateral sclerosis (ALS), and other neuromuscular diseases. The Association does this by funding worldwide research; by providing comprehensive healthcare services and support to MDA families nationwide; and by rallying communities to fight back through advocacy, fundraising, and local engagement.
The organization is funded by individual private contributions and cooperating organizations, providing research, services, and education. Currently, over 250 research projects are funded through the MDA. Although a national group, the MDA makes its resources and information accessible through local chapters, with 200 clinics throughout the states and 100 local offices. Some 44 of those clinics are MDA/ALS centers. MDA holds annual biannual clinical and scientific conferences where cutting-edge research and clinical trial information is presented. In addition, around 3,800 children attend MDA summer camp every year, and MDA’s online Transitions Center is a clearinghouse for resources to support young adults seeking employment, education, independent living, and community involvement; blogs are posted twice weekly from young adults sharing their experiences living with neuromuscular disease. MDA also hosts 150 support groups across the country. MDA’s advocacy program, based in Washington, DC, seeks to make the MD community’s voice heard and to expand resources for those with neuromuscular disease. By informing and educating legislators, MDA aims to accelerate development of new therapies. Individuals can sign up to be an MDA advocate and receive regular advocacy updates on the organization’s web site.
The MDA publishes a quarterly magazine, Quest Magazine, which is sent free of charge to families registered with MDA and is also archived online at http://quest.mda.org. The organization also has an online-only MDA/ALS Newsmagazine at http://alsn.mda.org. MDA is also active through social media channels, including Facebook and Twitter, enabling patients and caregivers to connect with one another and providing the latest research updates.
Duchenne MD
Parent Project Muscular Dystrophy (PPMD) focuses on finding an end to Duchenne MD specifically. In total, this group has invested over $45 million dollars in research, which has leveraged over $500 million in additional funding. PPMD has been involved in several steps forward for the MD community, including providing the FDA with the first-ever patient-initiated guidance to help accelerate development and review of potential therapies for Duchenne MD. PPMD also recently announced that a gene therapy study that it had funded might improve walking ability in MD. Historically, the group was instrumental in the passing of the MD Care Act in 2001. There are also a number of projects listed on their web site that need funding and lists of projects they have supported financially.
The PPMD web site has a helpful section, labeled as “Connect,” which provides links for the group’s monthly e-newsletter, updates on upcoming events, current research, and other MD groups. The organization has a mobile application for iPhone and Android users that informs patients of new clinical trials and other news, as well as location and contact information for nearby clinics. The PPMD Facebook and Twitter pages are regularly updated with pictures and news from the MD community, both scientific and patient-related. PPMD has also created a community page where visitors can post a profile in order to meet other members, read blogs, and share pictures. Many people who are affected by MD find that attending an event, having a chance to meet other patients or family members facing similar challenges can bring a sense of comfort and connectedness. The PPMD holds annual Connect Conferences and Meetings, typically attended by around 500 families.
There are other organizations keeping the end to Duchenne as a priority. CureDuchenne states on its website that it is a “national nonprofit that raises awareness and funds to find a cure for Duchenne muscular dystrophy.” This organization has helped fund two companies (Prosensa and Sarepta) that are seeking FDA approval for drugs for MD. CureDuchenne’s research investments have leveraged around $100 million from government agencies and pharmaceutical companies. In 2014, they hosted the sixth Climb for Duchenne, where “teams of people across the country can climb various mountains, hills, or tall buildings” to raise awareness and funds for CureDuchenne. The site encourages advocacy for fighting Duchenne by creating links for starting individual fundraisers.
The Foundation to Eradicate Duchenne (FED) was created in 2002 by Dana and Joel Wood, residing in Virginia, after their son was diagnosed with the disease. The Foundation’s web site says it has worked with others to achieve millions of dollars in federal earmarks for Duchenne MD research and a significant increase in the attention devoted to DMD at the National Institutes of Health. Additionally, through the FED and other fundraising efforts, the group has raised nearly $10 million in private donations and worked with Congress to secure nearly $40 million in federal appropriations. While not updated frequently, this group’s social network pages include several links for contact information from staff and for general questions, advocacy, and donations.
Related posts:
Duchenne and Becker Muscular Dystrophies: Underlying Genetic and Molecular Mechanisms
Transition from Childhood to Adult in Patients with Muscular Dystrophy
Overview of Current Treatments for Muscular Dystrophy
Key Challenges to the Approval of Products to Treat Patients with Muscular Dystrophy
Global and National Patient Registries
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