April 2024

As I’ve reflected on the incredible journey of PPMD these past few weeks, I am overwhelmed with gratitude for each and every one of you who have supported us over three decades. From the founding of PPMD in Cincinnati 30 years ago to the present day, our unwavering commitment, passion, and resilience have been the…

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PPMD is excited to announce a $250,000 award to Bo Hoon Lee, MD, from the University of Rochester to support  the development of a clinical research network for Duchenne babies identified through newborn screening efforts in New York State (NYS). The initiative aims to support newborn screening implementation efforts, inform clinical care guidelines for young…

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On April 19th and 20th, PPMD launched our brand new PPMD Together meeting series in Cincinnati, Ohio. The date and location held immense significance as we gathered to commemorate 30 years since the incorporation of PPMD in this very city in April 1994. Reflecting on 30 years of progress, our PPMD Together: Cincinnati meeting was…

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Most people with myotonic dystrophy type 1 (DM1) develop heart problems, according to a Danish study that followed patients for about 10 years. “Life-long cardiac screening is crucial, and we recommended that repeated follow-up is performed,” its researchers wrote in “Natural history of cardiac involvement in myotonic dystrophy type 1 – Emphasis on the need for lifelong follow-up,” which…

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I have three sons with Duchenne muscular dystrophy (DMD). Max, 18, Rowen, 15, and Charlie, 13, are part of a bustling family, joined by four other siblings: Lexi, 22, Chance, 16, Mary, 9, and Callie, 2. We have no sets of twins or multiples, yet I have often grouped together Max, Rowen, and Charlie. Max…

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Southern California’s Premier Wine Event Brought Together Acclaimed Napa Valley Vintnersand Philanthropists to Drive Research for Duchenne Muscular Dystrophy NEWPORT BEACH Calif., April 24, 2024 – CureDuchenne and Vintner Chair Alpha Omega hosted the Tenth Annual Napa in Newport on Saturday, April 20, 2024, at Pendry Newport Beach, which raised more than $1.35 million to find and fund a cure for Duchenne…

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In my last column, I wrote about the increasing difficulties I’m having safely navigating my home using the “furniture surfing” technique. I wondered whether it was time that I break out the walker and give it another try. I also mentioned my plans to contact my doctor to discuss starting physical and occupational therapy. A…

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Glucocorticoid use can help maintain breathing ability and arm function in adults with Duchenne muscular dystrophy (DMD), a new study reports. Importantly, the study found that glucocorticoid treatment was shown to be helpful even when only considering the time after patients had lost the ability to walk — which commonly occurs in the later stages…

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Capricor Therapeutics, which received early funding from CureDuchenne, has shared news of positive interactions with the FDA for their CAP-1002 program for DMD.  Capricor will be having an additional meeting with the FDA in Q2 2024, where they will share data and discuss the plan for submitting a rolling BLA, which might expedite their path…

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PPMD and Duchenne UK are excited to announce the recipient of their Joint Call for Therapeutic Projects. Pediatric neuromuscular neurologist Professor Peter Kang MD, from the University of Minnesota Medical School, has been awarded the grant for his research project titled “Phase 1 clinical trial of myogenic progenitors for DMD.” The grant, totaling $500,000, will…

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