Rare Disease Report

Advocacy Spotlight: Muscular Dystrophy Association

MAY 25, 2016
The Muscular Dystrophy Association (MDA) is a nonprofit association dedicated to improving the lives of patients with muscular dystrophy, amyotrophic lateral sclerosis (ALS), and related diseases. Supporting families lies at the heart of MDA’s mission. The top-rated charity fulfills this mission by funding research breakthroughs, caring for patients with these conditions, and empowering families through services and support. 

About neuromuscular diseases

Neuromuscular diseases include a broad range of medical conditions. Some are genetic muscular diseases, like Becker muscular dystrophy, that cause muscles to lose their structural integrity over time. Others result from metabolic disease which can affect many systems of the body, including the muscles. Others are neuromuscular diseases affecting the nervous system. For example, ALS causes problems with the motor neurons that control movement.
All neuromuscular diseases impair muscle movement in some way, causing muscle weakness and often paralysis. Depending on the disease, certain muscles and movements are affected first. Many neuromuscular diseases also have specific additional symptoms in other systems of the body. In some diseases only voluntary muscles are affected, but in others muscles such as the heart and diaphragm are also affected. Because of this, some neuromuscular diseases are eventually fatal.
Neuromuscular diseases can affect children and adults and males and females of all ethnic backgrounds. For some neuromuscular diseases, a single genetic cause is known and identified. Others may have a genetic component, but do not result from a single gene mutation. Most neuromuscular diseases do not have specific FDA approved treatments, but clinical research is very promising in some areas.

About MDA

In 1950, a patient with muscular dystrophy, Paul Cohen, founded what became the MDA. The organization came to be well known through its series of telethons which took place from 1956 to 2014, hosted by renowned comedians and entertainers.
Funding research is a cornerstone of the MDA’s mission. Through tackling multiple neuromuscular diseases, the MDA can often apply progress in one disease to other related conditions. MDA also helps promote research and collaboration through its annual clinical conference.
Currently, the organization is funding about 200 research projects in 12 countries. Just in 2015, MDA awarded grants for over $21.5 million. Thanks in part to MDA’s long-term research funding, dozens of clinical trials are now in progress for neuromuscular diseases, and advocates are hopeful this will soon translate into life-changing benefits for patients.
MDA also focuses on providing the best possible care available to families right now. Through its more than 150 Care Centers across the country, the MDA provides comprehensive care from a team of health specialists.
Providing family support is also key to the MDA mission. MDA provides an extensive network of support groups and educational activities for patients and family members. Through their summer camp, MDA provides thousands of children access to a free week of camp designed to support their needs. MDA also provides some families with financial assistance, such as for medical equipment. Through its world-class website, MDA provides informational resources to millions of people a year.
MDA has never shied away from big goals. Specifically, by 2020 MDA is aiming to double its research funding on drug development and clinical trials, provide care and support for an additional 50,000 families (double the current number), and fund 20,000 children at MDA’s summer camp.
Though much remains to be done in fighting for patients with neuromuscular diseases, MDA is leading the way. As one parent put it, “MDA means hope. It means strength. It means courage. MDA has been great helping [our son] overcome everyday limitations, and I can’t be more thankful.”

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