On Jan. 14, 2025, the U.S. Food and Drug Administration’s Center for Drug Evaluation and Research (CDER) announced…
On Nov. 11, 2024, an international team of researchers has for the first time cured patients with Toxic…
On Oct. 3, 2024, the National Health Service England (NHS) announced that Hundreds of babies have begun to…
On Oct. 3, 2024, scientists announced the largest whole-exome sequencing study of epilepsy to date, with more than…
On Sept. 25, 2024, a study led by scientists at Harvard Medical School announced they had developed an…
On Sept. 19, 2024, the World Health Organization (WHO) announced that Jordan had become the first country in…
On Mar. 13, 2024, the National Science Board (NSB) published The State of U.S. Science and Engineering 2024,…
On Jun. 29, 2023, the U.S. Food and Drug Administration approved Roctavian, an adeno-associated virus vector-based gene therapy…
On Mar. 13, 2023, the U.S. Food and Drug Administration (FDA) announced that it had approved Acadia Pharmaceuticals’…
On May 19, 2021, Megan A. Cooper, MD, PhD, an associate professor of pediatrics at Washington University School…
On Apr. 26, 2021, Sanofi has entered into an agreement with Moderna, under which Sanofi will help manufacture Moderna´s…
On Nov. 23, 2020, the U.S. Food and Drug Administration (FDA) approved Alnylam Pharmaceuticals’s Oxlumo (lumasiran) as the…
On Sept. 30, 2020, the National Institutes of Health (NIH) announced funding of a nationwide study to discover…
On May 7, 2020, the National Institutes of Health (NIH) announced a new online survey launched by the…
On Apr. 10, 2020, the U.S. Food and Drug Administration (FDA) approved selumetinib (KOSELUGO, AstraZeneca) for pediatric patients, 2…
On Mar. 31, 2020, researchers at the National Institutes of Health announced they had discovered a second gene…
On Aug. 15, 2019, the Cancer Prevention and Research Institute of Texas (CPRIT) announced that it was awarded…
On Feb. 25, 2019, the Tyrosinemia Society (TS, Inc.) was founded by Dr. Elizabeth Barnby, a researcher and…
On Aug. 15, 2017, the National Institutes of Health’s (NIH) Gabriella Miller Kids First Pediatric Research Program awarded…
On Jun. 17, 2014, Grifols opened state-of-the-art North Fractionation Facility (NFF) in Clayton, NC, where production capacity of…
On May 16, 2012, the U.S. Senate unanimously passed a resolution (S. Res. 286) recognizing May 16, 2012,…
On Mar. 2, 2008, Adult stem cells may provide an explanation for the cause of a Hutchinson-Gilford Progeria…
On Feb. 28, 2008, Rare Disease Day (also known as an “orphan” disease) is a globally coordinated movement…
On Sept. 24, 2007, David H. Murdock donated $35 million to Duke University to fund the Measurement to…
In 2001, the Stephenson Cancer Center was established. Located on the University of Oklahoma (OU) Health Sciences Center…
On Oct. 25, 1996, the first gene therapy trial for patients with Hunter syndrome was begun at the…
In 1987, the U.S. Food and Drug Administration (FDA) announced revised regulations regarding Expanded Access (EA) to investigational…
On Jan. 3, 1983, the Orphan Drug Act was passed by the U.S. Congress with the leadership of…
On Jun. 5, 1981, Dr. Michael Gottlieb and colleagues of University of California at Los Angeles reported a…
In 1976, the U.S. Centers for Disease Control and Prevention (CDC) was involved in a national immunization program…