U.S. Department of Defense Announces Duchenne Muscular Dystrophy Research Programs and Funding Opportunities for Fiscal Year 2025
We extend our deepest gratitude to the Members of Congress who fought to preserve Duchenne funding and to the Department of Defense for recognizing...
Three Ongoing Clinical Trials of Elevidys Gene Therapy Temporarily Halted in Europe
Three ongoing clinical trials of ELEVIDYS (delandistrogene moxeparvovec-rokl), a gene therapy developed by Sarepta and marketed by Roche, have been temporarily halted by the...
Translarna (Ataluren) Rejected by EMA: Marketing Authorization Not Renewed
In May 2024, PTC Therapeutics had been announced that the European Commission (EC) has decided not to adopt the CHMP's negative opinion on the...
Wave Life Sciences Announces Positive Data from FORWARD-53 Clinical Trial in DMD Including Significant Functional Benefit and Reversal of Muscle Damage after 48 Weeks...
Wave Life Sciences, a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced positive data...
Entrada Therapeutics Receives Authorization in the United Kingdom to Initiate ELEVATE-45-201
Entrada Therapeutics, announced it has received authorization from the United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) and Research Ethics Committee to initiate...