Rare Disease
Why Clearer Rare Disease Definitions Are Necessary To Creating Cures
Having a rare disease means you could have one of any of 5,000-7,000 distinct disorders, each with their own quirks. Just how many there are
Rare Patient News. Well Done.
Recent Advances put a Cure for Cushing’s Disease in Striking Distance
- February 21, 2020
Elderly Patients with Myelodysplastic Syndromes can Have Successful Transplants
Four Tips for Coping with a Chronic Illness
Can PharmaCyte’s “Cell-in-a-Box” be a Breakthrough for Pancreatic Cancer?
LATEST PATIENT WORTHY ARTICLES
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Rare Disease
LANDMARK STUDY: Scientists Analyzed the Genetic Code of 2,658 Cancer Genomes
Scientists in four continents worked for over a decade on a massive project that has resulted in the most detailed picture of cancer ever produced.
Rare Disease
Expanding AAV Manufacturing Capacity is Essential to Our Ability to Cure More Patients Living with Rare Disease
The National Center for Advancing Translational Sciences (NCATS) of the National Institutes of Health (NIH) and the Center for Biologics Evaluation and Research (CBER)
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Beta Thalassemia
Gene Therapy: The New Frontier for Cancer
Gene therapy is the treatment of disease by way of the transfer of genetic material into cells. According to a recent article in Labiotech,
Rare Disease
Rett Syndrome Drug Receives Fast Track Status
As originally reported in Financial Buzz, Rett Syndrome has received promising news: a drug undergoing review, ANAVEX®2-73 (blarcamesine), has received fast track designation, meaning patients
Bile duct cancer
Could This Investigational Drug be the Latest Advancement for Cholangiocarcinoma?
According to a story from curetoday.com, Dr. Tara Seery is an investigator in a phase 3 trial called PROOF, and she has just one goal:
