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Wrestling videos were the soundtrack to my morning earlier this week. I was staring at a beige wall void of anything except a small white clock marking the time and the black screen of a television no one uses. Sitting in the infusion center at our local hospital, I thought about how much time my…

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Most children with Duchenne muscular dystrophy (DMD) start experiencing notable issues with arm function and breathing ability before they lose the ability to walk, a new study reports. The findings have major implications for clinical trials testing treatments that aim to boost arm and/or lung function in DMD. Traditionally, such studies have enrolled only patients…

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It’s important to ensure that even within the context of Duchenne/Becker, kids are doing things that kids should be doing, adults are doing things that adults should be doing, and families can lead the lives they want to lead. Together with PPMD, your community, and your care team, it’s all about figuring out how we…

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 First nonsteroidal treatment approved for patients six years of age and older with DMD regardless of genetic mutation now available in the U.S.  ITF also announces the launch of ITF ARC (Access, Resources and Care) patient services program to bring suite of support and educational resources to patients and their families  Read the Full Press…

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PPMD is pleased to learn that DUVYZAT (givinostat) is now commercially available in the United States. DUVYZAT is a histone deacetylase (HDAC) inhibitor indicated for individuals diagnosed with Duchenne from six years of age and older that is being made available and marketed in the U.S. by ITF Therapeutics LLC. The drug was approved by…

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The U.S. Food and Drug Administration (FDA) has given the green light for Myogenica to start a clinical trial testing its stem cell therapy MyoPAXon in people with Duchenne muscular dystrophy (DMD). DMD, the most common type of muscular dystrophy, is caused by mutations in the gene that encodes the protein dystrophin. This protein normally…

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A few years ago, my wife, Wendy, and I took a mini-vacation to Philadelphia with my brother Ron, his wife, Judy, my brother Tim, and his wife, Diane. Ron, Tim, and I had visited relatives there on a regular basis growing up, so we cruised around the old neighborhoods where aunts and uncles used to…

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The approved therapy landscape has changed drastically in Duchenne, especially over the last year. With multiple newly approved therapies comes a lot of questions about how to navigate these drugs: when they are prescribed; who are the right patients for each drug; and how to navigate this ever-evolving and increasingly complex landscape. During the “Navigating…

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Thank you to everyone who helped us celebrate three decades of progress, community, and resilience at PPMD’s 30th Annual Conference, whether you joined us in person in Orlando, or tuned in virtually! PPMD’s 30th Annual Conference brought together our largest gathering of over 1,300 in-person and virtual attendees from all around the world. The three-day…

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READ THE FULL PRESS RELEASE HERE Introduction The journey towards finding effective treatments for Duchenne Muscular Dystrophy (DMD) has been marked by significant milestones. However, recent findings indicate that we need to re-evaluate and expand the Duchenne clinical trial enrollment criteria to serve the diverse DMD community. A study co-funded by CureDuchenne and the Collaborative…

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