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Celebrating 25 Years of Impact: MDA’s Wings Over Wall Street Gala Honors Leaders in ALS Research and Advocacy
For a quarter of a century, MDA’s Wings Over Wall Street has brought the ALS community together in New York City, raising millions of dollars to advance research and improve care. This year marks the 25th anniversary of the annual benefit, an evening dedicated to honoring extraordinary individuals who are driving progress in the fight…
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PPMD Hosts Duchenne Early Intervention Symposium
This May, the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) plans to vote on whether to add Duchenne to the Recommended Uniform Screening Panel (RUSP) for newborn screening. To support a push for evidence about the impact of early diagnosis on outcomes prior to the vote, PPMD recently hosted the Duchenne Early…
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On a rough day, insight into my sons’ life with DMD, wheelchairs
My husband, Jason, and I are parents to seven children: Lexi, 23; Max, 19; Chance, 17; Rowen, 16; Charlie, 14; Mary, 10; and Callie, 3. This week we’re traveling with five of them: Max, Rowen, and Charlie, who are our three sons with Duchenne muscular dystrophy (DMD), and our two youngest daughters. We’re visiting the…
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The Impact of the Continuing Resolution and Federal Budget Cuts to the CDMRP on Duchenne Research
Today, the Senate passed a Continuing Resolution (CR) that includes a 57% cut to the Congressionally Directed Medical Research Program (CDMRP). While the CR may keep the government running in the short term, it brings with it concerning implications for Duchenne and Becker muscular dystrophy research. The CDMRP, specifically the Peer-Reviewed Medical Research Program (PRMRP),…
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MDA Ambassador Guest Blog: Make Sense? Say Yes!
Grace LoPiccolo, 21, of St. Louis, Missouri, was diagnosed with CMT-1A at the age of nine. She currently attends Saint Louis University majoring in Bioethics and Health Studies, and Catholic studies. She is currently a Junior but ultimately plans on attending law school and pursuing a field in health and disability policy. Additionally, at Saint…
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Focus of NORD study: Rare disease community’s lived experiences
The National Organization for Rare Disorders (NORD) is seeking participants for its survey-based study Living Rare, which aims to better understand the real-world lived experiences of people in the U.S. with rare diseases. Living Rare, the first large-scale study of its kind in the U.S., seeks to capture the changing unmet needs and challenges faced by…
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My disease progression feels like barreling down a hill with no brakes
When I was 13 and my brother, Tim, was 16, we got skis for Christmas. I’m not sure why Mom and Dad decided to get them for us. Jeannette High School, my alma mater, had a ski club, but Tim and I weren’t members. I don’t recall asking for skis. Perhaps Tim did. However it…
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AB-1003 trial for LGMD proceeds after positive recommendation
Asklepios Biopharmaceutical (Askbio) has dosed the first participant in the second group of a Phase 1/2 trial testing AB-1003, an experimental gene therapy for limb-girdle muscular dystrophy (LGMD) type 2I/R9. The Phase 1/2 LION-CS101 trial (NCT05230459) is assessing AB-1003’s safety and tolerability in adults with LGMD2I/R9. Enrollment of the second group, which is ongoing at…
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Five Tips to Increase Socialization this Spring
Increasing socialization, cultivating meaningful relationships and friendships, and limiting isolation or loneliness in an increasingly remote world is a desire – and challenge – for many adults. Individuals living with disabilities sometimes face additional barriers of physical accessibility and social inclusion in their pursuit of a healthy and fulfilling social life. Add to that a…
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Advocates Unite in Washington D.C. to Champion Duchenne Policy Reforms
PPMD, with a group of more than 150 advocates, heads to Capitol Hill today to meet with Congressional leaders during this year’s annual PPMD Advocacy Conference. This marks the 23rd year that PPMD advocates will gather in Washington to urge Congress to advance legislation that will bring us closer to ending Duchenne. “Our community has…
