Celebrating Rare Disease Day
MDA families represent more than 40 different rare diseases—as all of the disorders under MDA’s umbrella are classified as “rare.”
Strongly
An expression of the live unlimited spirit, stories and voices of the MDA community
Building a Better Future with Shamrocks: Meet Shamrockstar Natalie, the Budding Architect
Over the past 35 years, the MDA Shamrocks program has raised more than $300 million to help save and improve the lives of families living with muscular dystrophy. Get to know Natalie, who graces the 2017 green MDA Shamrock with her sister Avery.
FirST-4-LEMS Study Seeks Participants
Researchers are looking for people with Lambert-Eaton myasthenic syndrome (LEMS) to participate in a phase 3 clinical trial, being conducted by Catalyst Pharmaceuticals, to test the investigational drug amifampridine phosphate. The Firdapse Strength Trial for LEMS, or FirST-4-LEMS, study is designed to evaluate the effectiveness of amifampridine phosphate in controlling, reducing and/or eliminating symptoms of . . .
No Mountain Too High: Woman With CMT Climbs Mount Kilimanjaro
CMT doesn’t hold Nicki back; it pushes her to reach higher. Literally. Read on to follow Nicki on the trek of a lifetime — to the top of Mount Kilimanjaro, the tallest mountain in Africa.
From Puerto Rico to Summer Camp: Meet Alejandro, the Shamrockstar who Found Hope Thanks to MDA
Alejandro, who is the local Atlanta-area ambassador for MDA, is one of ten “Shamrockstars” who will be featured on MDA Shamrocks this year at retailers across the country. His gold shamrock will be available exclusively at Lowe’s Home Improvement stores.
An MDA Summer Camp Love Story
Looking back, neither one of us went to camp expecting to meet the person we’d one day marry. … I can’t imagine my life without Zach. We can’t imagine our lives without our camp family or MDA Summer Camp.
Marathon Pharmaceuticals Pauses Commercialization of EMFLAZA to Address Concerns of Duchenne Community
Today, Marathon Pharmaceuticals, maker of the recently FDA-approved drug EMFLAZA, announced that they are pausing commercialization efforts in order to address the concerns about price and access to the drug from the Duchenne muscular dystrophy community. MDA will update our community with more information as it becomes available. We remain committed to ensuring that our families have the resources and assistance to begin and maintain treatments with emerging therapies for neuromuscular diseases.
FDA: Emflaza is Approved to Treat DMD in the United States
The FDA has approved Marathon Pharmaceuticals’ new drug applications for deflazacort (brand name Emflaza) to treat Duchenne Muscular Dystrophy
FDA Approves Emflaza for Treatment of Duchenne Muscular Dystrophy
The U.S. Food and Drug Administration has granted approval for deflazacort (brand name Emflaza), under development by Marathon Pharmaceuticals, to treat DMD
A Strong Voice: Meet Torrance, the Outspoken Shamrockstar
Over the past 35 years, the MDA Shamrocks program has raised more than $300 million to help save and improve the lives of families living with muscular dystrophy. Get to know Torrance, who graces the 2017 gold MDA Shamrock.
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