Strongly


An expression of the live unlimited spirit, stories and voices of the MDA community

Amicus Therapeutics Seeks Participants for Observational Pompe Disease Study

Researchers at Amicus Therapeutics are looking for participants with late-onset Pompe disease who are being treated with enzyme replacement therapy (ERT) to participate in an observational study. The study is designed to help researchers evaluate the change in muscle function and respiratory endpoints over approximately nine to 12 months. During that time, participants will have . . .

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Trial to Test Firdapse in MG Seeks Participants

Researchers are looking for people with myasthenia gravis (MG) to participate in a phase 3 clinical trial to evaluate the safety and effectiveness of the investigational drug amifampridine phosphate (brand name Firdapse). Effects of the drug will be assessed in individuals with muscle-specific kinase (MuSK) antibody positive MG and in a smaller group with acetylcholine . . .

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MLB Player Rhys Hoskins Hits One Home for MDA

Since his call-up to the Philadelphia Phillies in August 2017, Rhys Hoskins has taken Major League Baseball by storm. A prolific hitter, Rhys burst onto the scene late last summer with a home run barrage that made national news and shattered rookie records along the way. In just 10 months he has become the face . . .

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Trial to Test Drug Targeting Muscle Cramps in CMT Seeks Participants

Researchers are looking for people with Charcot-Marie-Tooth disease (CMT) to participate in a phase 2 clinical trial to evaluate the safety and effectiveness of the investigational drug FLX-787-ODT. FLX-787-ODT, under development by Flex Pharma, is taken in tablet form and is designed to reduce muscle cramps in adults with CMT. Trial length is approximately three . . .

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Trial to Test MG Drug UCB7655 Seeks Participants

Researchers are looking for people with myasthenia gravis (MG) to participate in a multinational phase 2A clinical trial to evaluate the safety, tolerability and effectiveness of the investigational drug UCB7665. UCB7665, under development by UCB Pharma, is administered via subcutaneous (under-the-skin) injection and is designed to reduce the severity of muscle weakness and fatigue in . . .

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On May 11, Drink One for Dane

On May 11, your daily cup of coffee can boost more than your energy — it can also bolster MDA’s quest to end ALS. For 12 years, Dutch Bros. Coffee has supported MDA through Drink One for Dane Day, a fundraiser launched in 2007 in honor of Dutch Bros. co-founder Dane Boersma, who was diagnosed . . .

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