The Muscular Dystrophy Association has been re-energized. What, you may ask, would make this national nonprofit organization, benefiting muscular dystrophy and its related life-threatening diseases, renew its focus at this particular time? The MDA, which is now over 65 years old, is celebrating its recent successes with the Food and Drug Administration. In just the past four years, five medications have been approved to treat muscular diseases – drugs that have been proven not only to treat the symptoms but also, in some cases, to slow the progression of the diseases.
“These breakthroughs have revived our focus on research,” said Rachel Crescibene, development director with the MDA in Greenville. “It was like getting wins back-to-back.”
With more than 40 different neuromuscular diseases under the muscular dystrophy umbrella, the MDA is the nation’s largest nonprofit supporter of muscle-debilitating diseases, that rob people of physical strength, independence and life. The five drugs approved by the FDA help with the fight against spinal muscular atrophy; amyotrophic lateral sclerosis, which is more commonly known as Lou Gehrig’s disease; and myasthenia gravis. Two of the drugs support patients with Duchenne muscular dystrophy.
The MDA’s excitement over the new approved medications is warranted because, often, “one finding could benefit another and then another and so on,” Crescibene said.
As the world leader in muscular dystrophy research, the MDA knows that when there are successes in research, there are successes with care. The organization supports families in so many ways, but its care centers for both children and adults provide a level of expert clinical care and medical research that is unmatched anywhere else for people suffering with muscular dystrophy, ALS and other neuromuscular diseases. In the Greenville area, there is a care center for adults and children at Greenville Hospital.
“We support 820 families in the Upstate alone. We offer everything from support groups to educational events and, most importantly, we connect these families to each other,” Crescibene said.
The Greenville area’s MDA offers local camps and medical equipment and plans various events throughout the year, such as the Harley Davidson Motorcycle Ride in April and the Muscle Walk in September.
“Also, about 40 fire departments participate in the Fill the Boot program in the spring and fall to help raise money for the MDA,” Crescibene added.
Since the national organization’s inception in the 1950s, the MDA has raised more than $1 billion for research on therapies and cures for these debilitating diseases.
“Seeing successes in research gives us that boost to continue our efforts to do more,” Crescibene said. “There is always more that we can do.”
For more information on the MDA Greenville organization and what you can do to help, visit www.mda.org or call 513-708-4965.
Drugs Approved by the FDA in the Last Four Years:
- Spinraza for spinal muscular atrophy – approved in December 2016.
- Deflazacort for Duchenne muscular dystrophy – approved in February 2017.
- Radicava for ALS – approved in May 2017.
- Eteplirsen for Duchenne muscular dystrophy – approved in September 2016.
- Soliris for myasthenia gravis – approved October 2017.
By Theresa Stratford