WASHINGTON, Aug. 27, 2024 /PRNewswire/ — Marking a significant milestone in improving the lives of children affected by Duchenne muscular dystrophy (Duchenne), Parent Project Muscular Dystrophy (PPMD) proudly announces that Massachusetts has approved the addition of Duchenne to the state’s newborn screening panel. This addition comes after years of dedicated advocacy by PPMD, in collaboration with partners and families, to break down barriers to treatment and care for the Duchenne community.
The addition of Duchenne to the Commonwealth’s newborn screen panel was included as an amendment to the Maternal Health bill (H. 4999) that was signed Friday, August 23, 2024, by Governor Maura Healey, with a ceremonial signing taking place Monday, August 26. The bill will be enacted in 90 days and Massachusetts has 18 months to begin screening all newborns for Duchenne, meaning that screening is expected to begin by June 2026, if not earlier.
Massachusetts is now the fourth state, after Ohio, New York, and Minnesota, to approve newborn screening for Duchenne. This milestone represents a significant advancement in the broader effort to expand newborn screening for Duchenne to other states.
Pat Furlong, PPMD’s President and CEO, passionately emphasizes the significance of newborn screening in Massachusetts, stating, “Our mission at PPMD is to accelerate research, shape policy, demand the highest standards of care for every family, and ensure access to life-changing therapies. Newborns identified through the screening program will benefit from vigilant developmental monitoring, timely physical and speech therapy, genetic counseling, and carrier screening for relatives. With eight FDA-approved treatments for Duchenne—four of which are approved for use in infancy—and more promising therapies in development, we remain steadfast in our commitment to providing the best possible care when it matters most: early in a child’s life.”
This is another pivotal step forward for newborn screening for Duchenne. PPMD’s vision is to ensure that all babies in all states have the same opportunities for diagnosis and care. PPMD continues to work towards the inclusion of Duchenne on the federal Recommended Uniform Screening Panel (RUSP), promoting newborn screening for Duchenne in other states, and expanding additional Certified Duchenne Care Centers across the country.
Lauren Stanford, PPMD’s Director of Advocacy, acknowledges the remarkable impact of the Duchenne community in the passage of this legislation, stating, “We commend the Massachusetts legislature for taking this crucial step to ensuring that every baby born is protected from the irreversible consequences of a diagnostic odyssey with Duchenne. This will also allow timely interventions during critical therapeutic windows for those diagnosed, giving them the best chance for effective treatment.”
PPMD has been building the infrastructure for early identification of newborns with Duchenne—including a robust therapeutic pipeline, regulatory infrastructure, and clinical care network and guidelines—since its inception and preparing for the implementation of newborn screening for Duchenne for almost a decade.
To learn more about PPMD’s work in newborn screening, click here.
ABOUT PARENT PROJECT MUSCULAR DYSTROPHY
Duchenne is a genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) fights every single battle necessary to end Duchenne.
We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won eight FDA approvals.
Everything we do—and everything we have done since our founding in 1994—helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne at EndDuchenne.org. Follow PPMD on Facebook, Twitter, Instagram, and YouTube.
SOURCE Parent Project Muscular Dystrophy (PPMD)
