MDA’s 2020 Advocacy Plan

It might only be a month into 2020, but MDA’s advocacy team and grassroots advocates are already making their voices heard. We expect the next 12 months to be crucial on Capitol Hill, and we’ll be working tirelessly in Washington, DC, to transform the lives of people living with neuromuscular disorders. Here’s a sneak peek . . .

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New MDA Educational Programs for Clinicians Offer a Detailed View on Selected Topics in Neuromuscular Disease

MDA’s Medical Education department was established in response to the growing need for clinician education in neuromuscular disease (NMD). We are pleased to present four webinars that cover newborn screening (NBS) and early intervention, genetic testing, and best practices in multidisciplinary care. Our Medical Education department, together with key opinion leaders in the NMD field, . . .

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Join an MDA Engage Newborn Screening Webinar on Aug. 30

We believe in the power of community and the importance of building relationships with families going through similar experiences. If you are an individual living with a neuromuscular disease, we invite you and your loved ones to attend our MDA Engage Newborn Screening Webinar. It will broadcast live online on Friday, Aug. 30, from 11 . . .

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US House Passes Newborn Screening Reauthorization

Thanks in large part to the work of MDA advocates across the country, the US House unanimously moved yesterday to pass the Newborn Screening Saves Lives Reauthorization Act (HR 2507). Once enacted, this law will reauthorize the national newborn screening program for five years and make additional investment in order to modernize and make improvements . . .

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FDA Approves PTC Therapeutics’ Emflaza for the Treatment of DMD in Patients Between 2 and 5 Years Old

On June 7, the U.S. Food and Drug Administration (FDA) approved Emflaza (deflazacort) to expand its labeling to include patients with Duchenne muscular dystrophy (DMD) who are between 2 and 5 years old. Emflaza was approved by the FDA in February 2017 for the treatment of DMD in patients 5 years and older, making it the first drug approved . . .

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Congress is Considering a Law to Strengthen Newborn Screening: Help MDA Promote its Passage

On May 2, Reps. Lucille Roybal-Allard (CA-40), Mike Simpson (ID-02), Katherine Clark (MA-05), and Jaime Herrera-Beutler (WA-03) introduced the Newborn Screening Saves Lives Reauthorization Act of 2019, a key piece of legislation that fights to eliminate preventable newborn deaths and stave off the onset of symptoms associated with severe disabilities, including neuromuscular conditions. Read MDA’s . . .

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JAMA Neurology Publishes Article on Newborn Screening and Neuromuscular Disorders

Earlier this week, JAMA Neurology published an article co-authored by Muscular Dystrophy Association (MDA) staff members that addresses the role of newborn screening (NBS) in the field of neuromuscular disease. In the article, “Maximizing the Benefit of Life-Saving Treatments for Pompe Disease, Spinal Muscular Atrophy, and Duchenne Muscular Dystrophy Through Newborn Screening: Essential Steps,” the . . .

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Progress in Motion: MDA’s 2019 Clinical & Scientific Conference Heralds New Directions for Neuromuscular Disease Treatment

In April, MDA held its first ever combined Clinical & Scientific Conference, themed “Progress in Motion,” in Orlando Fla. Clinicians, scientists, policymakers, nonprofit, and industry leaders convened for a dynamic and informative five days in Orlando, Fla. With more than 1,200 attendees, 23 sessions, 136 presentations, and 300 posters, this was MDA’s largest conference ever. . . .

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Arkansas to Screen Babies for SMA

This week, Arkansas Gov. Asa Hutchinson signed into law a bill, HB 1074, that requires the state to add spinal muscular atrophy (SMA) to its newborn screening panel. SMA is the No. 1 genetic cause of death in infants, affecting approximately 1 in 10,000 babies. MDA was proud to advocate for the addition of SMA . . .

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