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 support Dr. Kang and his co-investigators in evaluating the safety and tolerability of muscle progenitor cells for Duchenne muscular dystrophy. In response to the evolving landscape of gene and cell-based therapies, PPMD and Duchenne ..read more

PPMD is excited to announce another significant milestone in Duchenne newborn screening: Ohio will be the first state to screen all babies for Duchenne! More than 129,000 babies are born in Ohio every year, and the state expects to identify 35 babies with Duchenne annually. Ohio was the first state, followed by New York and Minnesota, to approve newborn screening for Duchenne when Ohio Governor Mike DeWine signed HB 33 into law in July 2023. The state now becomes the first in the nation to implement newborn screening for Duchenne. Ohio has long been a hub of clinical care and research advances ..read more

PPMD is excited to mark its 30th anniversary at the launch of the PPMD Together meeting series, taking place April 19-20, 2024, in Cincinnati, Ohio. Founded three decades ago by a small group of passionate parents and grandparents within the Duchenne community, PPMD has grown into a beacon of hope, driven by the unwavering dedication of parents, families, and individuals living with Duchenne and Becker. In April 1994, Cincinnati served as the birthplace of PPMD, where a handful of determined families joined forces to confront the challenges posed by Duchenne. Led ..read more

Edgewise Therapeutics, Inc., has announced positive two-year topline results from the ARCH open label study of sevasemten (EDG-5506) in adults with Becker. Sevasemten is an orally administered small molecule inhibitor designed to prevent contraction-induced muscle damage in dystrophinopathies, including Becker and Duchenne. Results showed that during two years of sevasemten treatment, participants’ North Star Ambulatory Assessment (NSAA) scores stabilized, in contrast to functional declines reported across multiple Becker natural history studies. In addition, significant decreases in key bioma ..read more

For more than a decade, PPMD has been at the forefront of advocating for Duchenne newborn screening. In 2023, we expanded our ongoing work in the space by conducting a parent survey to build evidence and understanding of the benefits of newborn screening for Duchenne. The results of the study have been published in the International Journal of Neonatal Screening. Drawing from the experiences of newborn screening efforts in other conditions, it is clear that comparing outcome data from a younger sibling diagnosed at an earlier age with an older sibling diagnosed and treated at an older age is i ..read more

PPMD is proud to announce the launch of our first state-specific Duchenne Advocacy Day in Illinois! This initiative marks a significant milestone in PPMD’s efforts to advance legislative progress for Duchenne at the state level. PPMD has long been at the forefront of Duchenne advocacy efforts. Its annual Advocacy Conference stands as the longest-running advocacy event for a rare disease community in the United States, with over two decades of advocacy in Washington, D.C. Now, PPMD expands its advocacy efforts to the state level with the launch of its pilot state-specific advocacy initiative. A ..read more

Over 60 leading experts in genetics, pediatrics, neurology, and public health advocates have authored a consensus statement in support of implementing Duchenne newborn screening. Key points of the consensus statement include: Timely Intervention: Early screening allows for prompt intervention and therapeutic strategies, slowing disease progression. Reduced Diagnostic Delays: Screening eliminates diagnostic delays, reducing avoidable costs and ensuring timely access to treatment. Public Health Impact: Screening aligns with preventive medicine principles, promoting early detection and intervent ..read more

ITF Therapeutics recently joined PPMD for a webinar to provide the Duchenne community with an opportunity to meet members of the ITF Therapeutics team to learn more about their organization and next steps to support the availability of DUVYZAT. ITF Therapeutics outlined next steps in the launch of DUVYZAT, including work on insurance policy education and coverage, healthcare provider education, gathering community feedback on services and programs, transitioning clinical trial participants to commercial product, and commercial product availability, which ITF expects later this year. The compan ..read more

Following the FDA’s approval of DUVYZAT (givinostat) on March 21, 2024, ITF Therapeutics, LLC, the company responsible for producing and marketing DUVYZAT in the United States, today shared an update on the drug and related resources. ITF Therapeutics has launched the DUVYZAT website, which provides Indication and Important Safety Information, a comprehensive list of FAQs, full Prescribing Information, a Patient Medication Guide, as well as an opportunity for individuals to sign up for updates on DUVYZAT. ITF Therapeutics has indicated that they expect DUVYZAT to be available in Q3 of 2024 and ..read more

PPMD is excited to learn that the FDA has granted approval to DUVYZAT(givinostat), a histone deacetylase (HDAC) inhibitor indicated for individuals diagnosed with Duchenne muscular dystrophy from six years of age and older. DUVYZATwill be made available and marketed in the United States by ITF Therapeutics, LLC.  DUVYZAT works by targeting pathogenic processes to reduce inflammation and loss of muscle. DUVYZAT is the first nonsteroidal drug approved for Duchenne that is applicable regardless of the specific genetic variant. Currently, it is not indicated for the treatment of individuals a ..read more