Strongly


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

Who’s On Your MDA Care Center Team?: Dietitians and Nutritionists

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 . . .

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Everyone Invited to Participate in Survey About Improving Genetic Counseling

In order to make progress toward ensuring all future genetic counselors possess the necessary skills to improve the quality of services provided to families and individuals, researchers at the University of Texas Health Sciences Center in Houston are seeking participants for an online survey. Participants will be asked to share their opinion in order to help identify . . .

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Who’s On Your MDA Care Center Team?: Speech-Language Pathologists

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 . . .

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Amylyx Announces Positive Results From Phase 2/3 Study of AMX0035 to Treat ALS

On Sept. 3, Amylyx announced positive results from its phase 2/3 CENTAUR trial of the investigational therapy AMX0035 (a combination of the small molecules sodium phenylbutyrate and taurursodiol) for treatment of amyotrophic lateral sclerosis (ALS). The primary endpoint of the study, assessment of functional decline over 24 weeks using the Amyotrophic Lateral Sclerosis Functional Rating . . .

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Look & Feel Your Best During Stressful Times: Tips From a Disability Fashion Blogger

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 . . .

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Reporting Back on MDA’s First-Ever Virtual Hill Day

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 . . .

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FDA Accepts Sarepta Therapeutics’ New Drug Application for DMD Treatment Casimersen

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 . . .

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Who’s On Your MDA Care Center Team?: Pediatric Social Workers

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 . . .

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