Sarepta announced a voluntary and temporary pause on all shipments of delandistrogene moxeparvovec-rokl (Elevidys) in the United States (U.S.). Elevidys is a single-dose, adeno-associated virus (AAV) vector-based gene therapy indicated for select patients with Duchenne muscular dystrophy (DMD). This halt comes after a U.S. Food and Drug Administration (FDA) request, following three deaths, to voluntarily stop all shipments of Elevidys. In June 2025, Sarepta released a statement following a second reported death from acute liver failure in a pediatric male patient. The first death was reported in March 2025. Both deaths occurred in non-ambulatory patients with DMD who received Elevidys. As a result of the second death, Sarepta suspended shipments of Elevidys for non-ambulatory patients with DMD and initiated a process to evaluate an enhanced immunosuppressive regimen. The FDA investigated these deaths and stated the two cases occurred in patients who presented with elevated transaminases; these patients were hospitalized less than two months following treatment with Elevidys. Product labeling includes a warning/precaution on acute serious liver injury but does not address the potential for liver failure or death. Following this communication, an additional FDA statement was released announcing a clinical hold on Sarepta’s investigational gene therapy (SRP-9004) trials for limb girdle muscular dystrophy (LGMD) following a patient fatality. In conjunction with this clinical hold, the FDA also requested Sarepta voluntarily stop all shipments of Elevidys. Initially, Sarepta refused the FDA’s request; however, Sarepta later announced it was pausing shipment to allow for collaboration with the FDA to complete the Elevidys supplemental safety labeling process and to address any questions. The FDA has also revoked the platform technology designation for the company’s AAVrh74 platform as the Agency has determined preliminary evidence is inadequate to demonstrate the platform technology can be used by more than one drug without adverse safety effects. Furthermore, the FDA has notified Sarepta that the indication of Elevidys in DMD should be restricted to ambulatory patients. Sarepta has provided additional details on the LGMD clinical trial patient fatality for their investigational gene therapy SRP-9004. Per Sarepta, the patient who passed away was a 51-year-old non-ambulant patient with LGMD who was receiving SRP-9004 in a Phase 1 clinical trial; SRP-9004 was used to treat a different disease and was administered at a different dose using a different manufacturing process than Elevidys. The study participant who developed acute liver failure and subsequently passed away was not treated with Elevidys. The FDA has stated that Elevidys and SRP-9004 use the same AAVrh74 serotype, and the investigation into the risk of acute liver failure with potentially life-threatening outcomes after use of these gene therapies is ongoing, including investigating the need for more regulatory actions. 

Since late February 2025, the United States (U.S.) Centers for Disease Control and Prevention (CDC) has been providing weekly updates on national measles cases in the U.S.  As of July 22, 2025, there were 1,319 confirmed cases of measles in the U.S. Most cases have occurred in pediatric individuals (378 in children less than five years of age, 484 in those five to 19 years of age). The majority of cases (92%) have been among individuals with unvaccinated or unknown vaccination status. Johns Hopkins Bloomberg School of Public Health is monitoring the measles outbreak in real-time through their Center for Outbreak Response Innovation (CORI) and International Vaccine Access Center (IVAC). The IVAC measles tracker displays a map of the U.S. capturing both cumulative and recent cases and distinguishes between local and imported cases. Data are included from state and county health departments as well as news sources. The data represent laboratory-confirmed measles cases reported by public health authorities. The largest outbreak was in west Texas, and other outbreaks have spread from this area. IVAC also displays county and state level data, cumulative cases reported by year, percentage of reported measles cases by vaccination status and measles cases by age. CORI reports the total U.S. measles cases in 2025 on a dashboard that includes a map displaying geographic regions of the cases; the dashboard also includes the number of deaths from measles in the U.S., Canada and Mexico. Measles is an extremely infectious airborne virus that can lead to severe rash and complications, potentially including pneumonia, encephalitis and/or death due to respiratory or neurologic problems. It is especially dangerous for infants and children less than five years of age as well as pregnant individuals, adults > 20 years of age and those with weakened immune systems. Vaccination with a measles, mumps and rubella (MMR) or a measles, mumps, rubella and varicella (MMRV) vaccine is highly effective at prevention of measles. One dose of the MMR vaccine is 93% effective at prevention of measles; two doses are 97% effective. The CDC recommends routine childhood vaccination with two doses of the MMR or MMRV vaccine with the first dose administered between 12 to 15 months of age and the second dose given between four and six years of age.