An expression of the live unlimited spirit, stories and voices of the MDA community
At more than 150 MDA Care Centers nationwide, kids and adults living with neuromuscular disease (NMD) have access to multidisciplinary care teams made up of different specialists who can coordinate in one place on an appointment day to optimize a complete care plan. Included on these teams are allied health professionals and social workers who . . .
September is Newborn Screening Awareness Month, and today, Sept. 7, is World Duchenne Awareness Day, dedicated to spreading knowledge about Duchenne muscular dystrophy. As we honor both, we’re providing an update on our work to ensure every state in the US screens babies at birth for DMD, leading to improved outcomes and longer, stronger lives. . . .
Lainie Ishbia, MSW, is a mom, wife, blogger, fashionista, and disability and empowerment speaker. She wears leg braces and struggles with buttons and everyday fine motor tasks because of a slowly progressive neuromuscular disorder called Charcot-Marie-Tooth disease (CMT). Lainie is passionate about helping people with disabilities to look and feel their best. She created a lifestyle and . . .
When the COVID-19 pandemic made meeting in person impossible, MDA’s Advocacy program needed to shift from in-person to virtual meetings with lawmakers. In early August, volunteers met with their lawmakers virtually — for the first time — to urge elected officials in Washington, DC, to end the process of surprise medical billing, which can be . . .
At more than 150 MDA Care Centers nationwide, kids and adults living with neuromuscular disease (NMD) have access to multidisciplinary care teams made up of different specialists who can coordinate in one place on an appointment day to optimize a complete care plan. Included on these teams are allied health professionals and social workers who . . .
On Aug. 25, Sarepta Therapeutics announced that the US Food and Drug Administration (FDA) accepted the company’s New Drug Application (NDA) seeking accelerated approval for casimersen (SRP-4045), an investigational therapy for Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping. The FDA has granted Sarepta’s application Priority Review Status, meaning that a decision is expected . . .
At more than 150 MDA Care Centers nationwide, kids and adults living with neuromuscular disease (NMD) have access to multidisciplinary care teams made up of different specialists who can coordinate in one place on an appointment day to optimize a complete care plan. Included on these teams are allied health professionals and social workers who . . .
Today, the US Food and Drug Administration (FDA) granted accelerated approval to viltolarsen (Viltepso) for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. It is the third exon-skipping, disease-modifying drug to treat DMD, the most common childhood form of muscular dystrophy. The drug is administered by intravenous infusion. Viltepso will . . .
On Aug. 7, the US Food and Drug Administration (FDA) granted approval of risdiplam (Evrysdi) for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. It is the third disease-modifying therapy approved to treat SMA, the leading genetic cause of infant death. The drug is an orally administered liquid, the first medicine that could . . .
Josh Cueter, 22, from Troy, Michigan, is a 2020 graduate from Michigan State University. At school he was a board member of the MSU Adaptive Sports & Recreation Club as well as MSU’s Council of Students with Disabilities. After a semester internship with the Equal Employment Opportunity Commission in Washington, DC, Josh is currently working . . .
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