We are devastated to learn of the tragic loss of a young boy that was participating in Pfizer’s Phase 2 DAYLIGHT study. This study is evaluating the safety and dystrophin expression of fordadistrogene movaparvovec (PF-06939926), Pfizer’s gene therapy candidate for Duchenne, in boys who are two and three years old. According to the company, “We do not yet have complete information and are actively working with the trial site investigator to understand what happened.” Pfizer has indicated that the individual received the investigational gene therapy in early 2023. Additionally, “Initial dosing f ..read more

PPMD, in collaboration with the Muscular Dystrophy Association (MDA), the EveryLife Foundation for Rare Diseases, and the Little Hercules Foundation (LHF), recently hosted a webinar on the background, history, and next steps for Duchenne Newborn Screening on both the state and federal levels.  What is Newborn Screening? Annie Kennedy, Chief of Policy, Advocacy, and Patient Engagement for the EveryLife Foundation, provided a comprehensive overview of newborn screening, detailing what the process entails and what happens in the case of a positive or “out of range” screen.  She also e ..read more

PPMD is pleased to share that a new genetic counseling practice resource for dystrophinopathies, including Duchenne and Becker muscular dystrophy and carriers, has been published in the Journal of Genetic Counseling.  PPMD Certified Genetic Counselors, along with other board-certified genetic counselors from the National Society of Genetic Counselors (NSGC), served as authors of this practice resource, for which PPMD provided official endorsement. This practice resource aims to address the unique and complex aspects of genetic counseling for Duchenne and Becker muscular dystrophy and carr ..read more

We are thrilled to welcome the newest addition to the PPMD team, Jodi Wolff, PhD, who joins us as our Chief Program Officer! Jodi brings with her a wealth of experience and an unwavering passion for advocating for individuals with rare diseases, particularly those with neuromuscular conditions such as Duchenne and Becker muscular dystrophy. Jodi’s commitment to rare diseases has been longstanding and multifaceted. From her early adulthood roles as a camp volunteer for the Muscular Dystrophy Association (MDA), then as a social worker in pediatric and adult neuromuscular clinics, to her impressi ..read more

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 driving force behind our fight for every future. Thank you for honoring our milestone anniversary this past month. The proclamation in Cincinnati, the messages from so many of you on social media, and the recurring gifts made or increased have truly warmed my heart.   There ..read more

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 not just a commemoration but an earnest commitment to the dynamic needs of our community, bringing together families, industry partners, care providers, and advocates in a collaborative effort. On Friday evening, PPMD community members, including families, members of ..read more

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 patients, and lay the foundation for expansion of the network across states. Duchenne is the most common genetic condition diagnosed in childhood, affecting approximately one in 5,000 live male births. Early diagnosis is critical for ..read more

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