An advisory committee has postponed its decision to decide whether Duchenne muscular dystrophy should be included in a federally-recommended newborn screening panel for up to one year, Parent Project Muscular Dystrophy announced Thursday.
The next advisory committees will convene Aug. 8 and 9, and Nov. 14 and 15.
Parent Project Muscular Dystrophy (PPMD) — a Duchenne patient advocacy group — submitted the nomination package to add Duchenne muscular dystrophy to the Recommended Uniform Screening Panel, or RUSP for short. The panel is a list of disorders that the US Department of Health and Human Services recommends for newborn screening.
States, which regulate which conditions are screened, are not required to include the conditions from the RUSP, though many do. Only 12 states have adopted “RUSP alignment legislation,” according to the nonprofit group EveryLife Foundation for Rare Diseases, meaning that they are required to screen newborns for any disorders recommended by the federal panel.
The conditions added to the RUSP are typically those that can be treated if detected early on in a newborn’s life.
The vote by the advisory committee was initially planned for Thursday afternoon, but PPMD asked that the vote be moved back so it could put together additional data on the benefit of early detection of Duchenne muscular dystrophy. Researchers are putting together data on how early care and treatment with steroids could benefit children born with Duchenne, PPMD’s founder Pat Furlong, a parent of boys with Duchenne, told Endpoints.
HHS did not immediately return a request for comment.
The advocacy group initially submitted for inclusion on the RUSP in 2022, but the advisory committee asked for additional data. In 2023, PPMD resubmitted the Duchenne nomination.
If the advisory committee votes in favor of adding Duchenne to the RUSP, the decision would be sent to the HHS Secretary for final approval.
Editor’s note: This story has been corrected to reflect that the vote has been postponed for up to one year. A previous version stated the vote was postponed until November.