According to The Washington Post, on Tuesday the Food and Drug Administration, FDA, made history by approving a gene therapy for childhood blindness, which will change the lives of many…
As a scientist, Mary-Claire King has had a remarkable career related to her study of BRCA genes. She’s received a Lasker Award and has been recognized by President Obama with…
Great news for the rare disease community! Ultragenyx Pharmaceuticals reported back positive results from its phase 3 study of the treatment drug burosumab in adults with X-linked hypophosphatemia (XLH). XLH…
Our gut bacteria may be responsible for antiphospholipid syndrome (APS). What do you need to know about the ordinary gut bacteria known as commensals, and APS? Keep reading, or view…
Good news for patients with Mucopolysaccharidosis Type VII (MPS VII)! In mid-November, the FDA approved a new drug for treatment of this rare disorder. The drug can be used for…
Epidermolysis bullosa (EB) is a rare disorder, in which a genetic mutation disrupts the production of a protein that holds the skin together. This creates intense and painful blistering externally,…
Big news for those with hemophilia! On Thursday, the FDA approved Hemlibra (manufactured by Genentech), a weekly self-injection for hemophilia A patients who have developed resistance to standard medicines for preventing…
Vinmec, a healthcare facility in Vietnam made bronchopulmonary dysplasia (BPD) history. They successfully treated BDP using a method that had never been tried before: the stem cell transplant. The procedure occurred…
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