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.

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Social Workers: Powerful Members of Your Health Care Team

For individuals and families living with neuromuscular diseases, having access to the right support is paramount to living life to the fullest. Groundbreaking research, clinical trials and care from the best physicians are critical. But so is everyday help, and that’s where MDA Care Center social workers come in. Social workers can help individuals address . . .

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Why Multidisciplinary Care Matters

Clara was 2 years old when she was diagnosed with congenital muscular dystrophy. Her family was referred to their local Minneapolis MDA Care Center, and when they got there, Clara’s mom, Becky, says it felt “a bit like coming home.” Our MDA Care Center is a huge gift for us as working parents managing a . . .

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Got questions? MDA’s new Resource Center has answers

MDA is pleased to announce that we have launched a new MDA National Resource Center that is now available to provide families one-on-one support from trained specialists. You can contact our MDA specialists at resourcecenter@mdausa.org and 800-572-1717 for resources and support. Specialists are available Monday through Friday 8 a.m. to 5:30 p.m. CST and are typically . . .

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Marathon Pharmaceuticals Announces Pivotal Phase 3 Data for Deflazacort for DMD in Neurology

Marathon Pharmaceuticals has announced pivotal Phase 3 data evaluating the investigational drug deflazacort for the treatment of Duchenne muscular dystrophy in the journal Neurology. The study results show that deflazacort demonstrated significant improvement in muscle strength compared with placebo in 12 weeks. In addition, results show that deflazacort was associated with less weight gain than . . .

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“MissionDMD” Trial Seeks Participants

Researchers are looking for boys and men living with Duchenne muscular dystrophy, ages 12 years and older, to participate in a phase 2 clinical trial, sponsored by FibroGen, to test the experimental drug FG-3019 (pamrevlumab). The study, known as “MissionDMD,” is designed to help researchers evaluate whether FG-3019 is safe and effective in people with . . .

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Join Us for an Emergency Preparedness Webinar

September is National Preparedness Month. Emergencies can happen in an instant and without warning. From weather-related events and other natural disasters to emergencies such as chemical spills and power outages, the unexpected can put you and your family in danger — unless you plan ahead. Please join us for an important discussion about how families living . . .

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