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ITF Therapeutics LLC Announces U.S. Commercial Availability of DUVYZAT™ (givinostat) for Treatment of Patients With Duchenne Muscular Dystrophy
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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ITF Therapeutics Announces U.S. Commercial Availability of DUVYZAT™ (givinostat) for Treatment of Duchenne
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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FDA OKs clinical trial of DMD stem cell therapy MyoPAXon
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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I wish more facilities considered accessibility and privacy
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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Navigating the Approved Therapy Landscape
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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PPMD’s 30th Annual Conference Session Recordings
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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Expanding Duchenne Clinical Trial Criteria: A Crucial Step Forward
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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Breakthrough in skeletal muscle regeneration
In a finding that opens the door to the development of targeted therapies for various muscle disorders, newly published research identifies key mechanisms of skeletal muscle regeneration and growth of muscles following resistance exercise.
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How a son’s MRI stirred up fields of dreams and hopes
This week, one of my boys had to get an MRI. Three of my four sons — Max, 18, Rowen, 15, and Charlie, 13 — are living with Duchenne muscular dystrophy (DMD), and they’ve had annual MRIs for the past 14 years (well, fewer for Charlie). Sometimes they’ve had more than one a year because…
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CureDuchenne Celebrates FDA’s Launch of Rare Disease Innovation Hub
We at CureDuchenne are excited to acknowledge the recent announcement by the United States Food and Drug Administration (FDA) about the creation of the “Rare Disease Innovation Hub.” This new initiative represents a significant step forward in enhancing support and treatment options for rare disease patients, particularly those living with Duchenne and Becker muscular dystrophy.…
