This first-ever treatment is approved for individuals with Rett syndrome. (Updated March 19, 2023) Today, March 10, 2023, Acadia Pharmaceuticals announced that their investigational drug, Trofinetide, has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of Rett syndrome. The drug, marketed under the name DAYBUE™, is expected to be commercially … Read More
The fund is established thanks to an inaugural gift from the Otis Family in honor of their son, Barrett. The Rett syndrome research landscape has never been more exciting. New clinical trials are underway in gene therapy, and we eagerly await the FDA’s decision about Trofinetide. But we know that families of males with Rett … Read More
READ TAYSHA’S LETTER TO THE RETT COMMUNITY DALLAS, Jan. 31, 2023 (GLOBE NEWSWIRE) — Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, clinical -stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic rare diseases of the central nervous system (CNS), today provided an update on the TSHA-120 program in giant axonal … Read More
We’re excited to share that Neurogene announced today that the FDA approved their Investigational New Drug Application (IND) to launch a clinical trial for their gene therapy treatment NGN-401 in 2023 in pediatric females. This is the second gene therapy clinical study in Rett syndrome currently underway and the very first gene therapy clinical trial … Read More
By Dr. Tim Benke, IRSF Medical Advisor As many of you know, the United States is currently facing the triple impact of flu, RSV, and COVID-19. These respiratory illnesses are currently circulating at levels higher than usual for this time of year, creating a surge of patients seeking care in clinical offices, emergency rooms, and … Read More
