Experimental Compound Offers Potential Treatment for Rare, Often Fatal, Childhood Disease
On Jul. 9, 2025, as part of a new study, neurologists at NYU Langone Health announced they have…
Diagnostics | Disease | Infectious Disease | Medicine | Oncology | Rare Disease | Therapeutics | WHO
Global leaders reaffirm commitment to WHO with More Than US$ 170 million
On May 20, 2025, the World Health Organization (WHO) announced that world leaders pledged at least an additional…
Prime Medicine Announces Breakthrough Data Showing Rapid Restoration of DHR Positivity After Single Infusion of PM359, an Investigational Prime Editor for Chronic Granulomatous Disease
On May 19, 2025, Prime Medicine announced positive initial data from the first patient dosed in its ongoing…
World’s first patient treated with personalized CRISPR gene editing therapy at Children’s Hospital of Philadelphia
On May 15, 2025, a team at Children’s Hospital of Philadelphia (CHOP) and Penn Medicine announced that a…
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HHS starts layoffs of thousands of workers across its agencies
On Apr. 1, 2025, layoff notices began arriving for thousands of employees of the sprawling U.S. Department of…
Biotechnology | Disease | FDA | Rare Disease
FDA Approved Qfitlia™ (fitusiran), the First siRNA (RNAi Therapeutic) for the Treatment of Hemophilia A or B
On Mar. 28, 2025, the U.S. Food and Drug Administration (FDA) announced approval of Alnylam Pharmaceuticals Qfitlia™ (fitusiran),…
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75% of U.S. scientists who answered Nature poll consider leaving
On Mar. 27, 2025, a Nature survey of more than 1,200 scientists, three-quarters of the total respondents are…
Disease | FDA | Rare Disease
Alnylam Announces FDA Approval of AMVUTTRA® (vutrisiran), the First RNAi Therapeutic to Reduce Cardiovascular Death, Hospitalizations and Urgent Heart Failure Visits in Adults with ATTR Amyloidosis
On Mar. 20, 2025, Alnylam Pharmaceuticals announced the U.S. Food and Drug Administration (FDA) approval of the supplemental…
Diagnostics | Disease | FDA | Medical Device | Non-Profit Research | Ophthalmogy | Rare Disease | Therapeutics
FDA approves ENCELTO, a first-of-its-kind eye implant that slows vision loss in rare eye disease
On Mar. 6, 2025, the U.S. Food and Drug Administration (FDA) announced the approval of ENCELTO (revakinagenetaroretcel-lwey), a…
St, Judes Reports Promising results from first prenatal therapy for spinal muscular atrophy
On Feb. 19, 2025, scientists at St. Jude Children’s Research Hospital report results from a promising new approach…
Biotechnology | Diagnostics | FDA | Medical Device | Medicine | Pharmaceutical | Rare Disease | Therapeutics
U.S. Food and Drug Administration approved 50 new therapeutics in 2024
On Jan. 14, 2025, the U.S. Food and Drug Administration’s Center for Drug Evaluation and Research (CDER) announced…
World-first cure for lethal skin disease achieved in landmark study
On Nov. 11, 2024, an international team of researchers has for the first time cured patients with Toxic…
Biotechnology | Diagnostics | Disease | Genomics | Non-Profit Research | Rare Disease | Therapeutics | Women's Health
First newborn babies tested for over 200 genetic conditions as world-leading study begins in NHS hospitals
On Oct. 3, 2024, the National Health Service England (NHS) announced that Hundreds of babies have begun to…
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…
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 | 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…
Biotechnology | Diagnostics | Disease | FDA | Rare Disease | Therapeutics
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…
Biotechnology | COVID-19 | Disease | Infectious Disease | Oncology | Rare Disease | Therapeutics | Vaccine
Moderna announced agreement with Sanofi for manufacturing of Moderna COVID-19 Vaccine in the U.S.
On Apr. 26, 2021, Moderna announced that it had entered into an agreement with Sanofi for fill/finish sterile…
Disease | FDA | Rare Disease
FDA approved first drug to treat rare metabolic disorder
On Nov. 23, 2020, the U.S. Food and Drug Administration (FDA) approved Alnylam Pharmaceuticals’s Oxlumo (lumasiran) as the…
FDA approved first therapy for children with debilitating and disfiguring rare disease
On Apr. 13, 2020, the U.S. Food and Drug Administration (FDA) announced that given the increased demand for…
Biotechnology | Disease | Non-Profit Research | Oncology | Pharmaceutical | Rare Disease | Therapeutics
CPRIT granted $18.7 million New Company Product Development award to Salarius Pharmaceuticals
On Aug. 15, 2019, the Cancer Prevention and Research Institute of Texas (CPRIT) announced that it was awarded…
NIH selected OHSU Knight Cancer Institute to co-lead largest national database for pediatric cancer, rare disease research
On Aug. 15, 2017, the National Institutes of Health’s (NIH) Gabriella Miller Kids First Pediatric Research Program awarded…
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 | 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…