Clinical Trials During COVID-19: What We Need From the FDA

Over the course of the last several months, the US Food and Drug Administration (FDA) has undertaken many actions aimed at slowing the spread of the novel coronavirus and ensuring our first responders have the medical equipment, devices, diagnostic tools, and therapies to treat those with COVID-19. Simultaneously, the FDA has issued guidance on how . . .

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MDA Advocacy in These Uncertain Times

We know families across the country, especially those in the neuromuscular disease (NMD) community, are concerned about the spread of coronavirus and its associated disease, COVID-19, and the impact on their loved ones. In these uncertain times, MDA’s advocacy team will not stop serving and amplifying the voices of those in the NMD community, especially . . .

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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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Why Attending MDA’s Upcoming Pompe Disease PFDD Meeting is Vital

Ryan Colburn is a rare disease patient with a professional background in engineering and operations management, spending portions of his career working on racecars, airplanes, and rockets. Diagnosed with Pompe disease in 2015, he has spent the time since learning about rare disease topics including research, advocacy, and drug development to better understand how to . . .

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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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Congress Moves Forward on Newborn Screening

Last week, the US House Committee on Energy and Commerce’s Subcommittee on Health considered the Newborn Screening Saves Lives Reauthorization Act (HR 2507), a bill that would reauthorize the national newborn screening program for five years. We are happy to report that the subcommittee unanimously agreed to send the bill to the full committee for . . .

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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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