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…
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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…
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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…
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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…
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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…
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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…
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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…
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Biogen received European Commission approval for QALSODY® (tofersen), first therapy for rare, genetic form of ALS
On May 30, 2024, Biogen announced that the European Commission (EC) had granted marketing authorization under exceptional circumstances…
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Artificial Intelligence | Biotechnology | Cloning | CRISPR | Forestry | Genomics | Imaging | Infectious Disease | Marine Science | Medical Device | Medicine | Nutraceuticals | Oncology | Pharmaceutical | Quantum | Rare Disease | Stem Cell | Therapeutics | U.S. Congress | Vaccine
National Science Board published “The State of U.S. Science and Engineering 2024”
On Mar. 13, 2024, the National Science Board (NSB) published The State of U.S. Science and Engineering 2024,…
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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…
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FDA approved first treatment for Rett syndrome
On Mar. 13, 2023, the U.S. Food and Drug Administration (FDA) announced that it had approved Acadia Pharmaceuticals’…
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Scientists discovered rare genetic condition that attacks kids’ immune systems
On May 19, 2021, Megan A. Cooper, MD, PhD, an associate professor of pediatrics at Washington University School…
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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, Sanofi has entered into an agreement with Moderna, under which Sanofi will help manufacture Moderna´s…
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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…
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NIH funded first nationwide network to study rare forms of diabetes
On Sept. 30, 2020, the National Institutes of Health (NIH) announced funding of a nationwide study to discover…
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Biotechnology | FDA | Oncology | Rare Disease | Therapeutics
Ultragenyx and Kyowa Kirin Announce U.S. FDA Approval of Crysvita® (burosumab) for Treatment of Tumor-Induced Osteomalacia
On Jun. 18, 2020, Ultragenyx Pharmaceutical and Kyowa Kirin announced that the U.S. Food and Drug Administration (FDA)…
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FDA | Neurology | Ophthalmogy | Rare Disease
The FDA approved new therapy for rare disease affecting optic nerve, spinal cord
On Jun. 11, 2020, the U.S. Food and Drug Administration approved Viela Bio’s Uplizna (inebilizumab-cdon) injection for intravenous…
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NIH-supported research examines impact of COVID-19 on rare diseases community
On May 7, 2020, the National Institutes of Health (NIH) announced a new online survey launched by the…
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Biotechnology | FDA | Oncology | Rare Disease | Therapeutics
FDA approved first therapy for children with debilitating and disfiguring rare disease
On Apr. 10, 2020, the U.S. Food and Drug Administration (FDA) approved selumetinib (KOSELUGO, AstraZeneca) for pediatric patients, 2…
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Biotechnology | Genomics | NIH | Rare Disease | Therapeutics
NIH researchers discovered gene for rare disease of excess bone tissue growth
On Mar. 31, 2020, researchers at the National Institutes of Health announced they had discovered a second gene…
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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…
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The Tyrosinemia Society Was founded
On Feb. 25, 2019, the Tyrosinemia Society (TS, Inc.) was founded by Dr. Elizabeth Barnby, a researcher and…
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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…
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Grifols opened state-of-the-art facility in Clayton to advance treatment of rare and life-threatening diseases
On Jun. 17, 2014, Grifols celebrated the grand opening of its new state-of-the-art North Fractionation Facility (NFF) in…
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The U.S. Senate declared May 16 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,…
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National Cancer Institute reported changes in adult stem cells may underlie disease associated with accelerated aging
On Mar. 2, 2008, Adult stem cells may provide an explanation for the cause of a Hutchinson-Gilford Progeria…
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Rare Disease Day (also known as an “orphan” disease) was established
On Feb. 28, 2008, Rare Disease Day (also known as an “orphan” disease) is a globally coordinated movement…
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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…
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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…
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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…
