Exome sequencing of more than 54,000 individuals with epilepsy revealed genetic links
On Oct. 3, 2024, scientists announced the largest whole-exome sequencing study of epilepsy to date, with more than…
Artificial Intelligence | Biotechnology | Life Science News | Non-Profit Research | Rare Disease | Therapeutics
Researchers used AI to Repurpose Existing Drugs for Treatment of Rare Diseases
On Sept. 25, 2024, a study led by scientists at Harvard Medical School announced they had developed an…
Biotechnology | Infectious Disease | Life Science News | Medicine | Non-Profit Research | Rare Disease | Therapeutics | WHO | Women's Health
WHO verified Jordan as first country to eliminate leprosy
On Sept. 19, 2024, the World Health Organization (WHO) announced that Jordan had become the first country in…
U.S. FDA approved BioMarin’s ROCTAVIAN, the first and only gene therapy for adults with severe hemophilia A
On Jun. 29, 2023, the U.S. Food and Drug Administration approved Roctavian, an adeno-associated virus vector-based gene therapy…
Grifols opened state-of-the-art facility in Clayton to advance treatment of rare and life-threatening diseases
On Jun. 17, 2014, Grifols opened state-of-the-art North Fractionation Facility (NFF) in Clayton, NC, where production capacity of…
The U.S. Senate declared May 16, 2012 as Hereditary Angioedema Awareness Day (HAE)
ON May 16, 2012, the U.S. Senate unanimously passed a resolution (S. Res. 286) recognizing May 16, 2012,…
Rare Disease Day (also known as an “orphan” disease) was established
On Feb. 28, 2008, Rare Disease Day (also known as an “orphan” disease), was established by the European…
David H Murdock donated $35 million to Duke University to fund the Measurement to Understand the Reclassification of Disease
On Sept. 24, 2007, David H. Murdock donated $35 million to Duke University to fund the Measurement to…
Biotechnology | Diagnostics | Disease | Life Science News | NIH | Oncology | Rare Disease | Therapeutics
The Stephenson Cancer Center was established
In 2001, the Stephenson Cancer Center was established. Located on the University of Oklahoma (OU) Health Sciences Center…
First gene therapy trial for patients with Hunter syndrome began at the University of Minnesota
On Oct. 25, 1996, the first gene therapy trial for patients with Hunter syndrome was begun at the…
Investigational drug regulations revised to expand access to experimental drugs for patients with no alternative therapies
In 1987, the U.S. Food and Drug Administration (FDA) announced revised regulations regarding Expanded Access (EA) to investigational…
The Orphan Drug Act was passed by the U.S. Congress
On Jan. 3, 1983, the Orphan Drug Act was passed by the U.S. Congress with the leadership of…
national immunization program for swine flu and Guillain-Barre syndrome Cause is Unknown
In 1976, the U.S. Centers for Disease Control and Prevention (CDC) was involved in a national immunization program…
Biology | Diagnostics | Disease | Life Science History | NIH | Oncology | Rare Disease | Therapeutics
The U.S. government established a national blood collection program
In 1940, the U.S. government established a national blood collection program. That same year the National Research Council…