Tag Archives: disease

FDA Approves New Treatments Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) for Heart Disease Caused by Transthyretin Mediated Amyloidosis

Print this page May 06, 2019 — On May 3, the U.S. Food and Drug Administration approved Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) capsules for the treatment of the heart disease (cardiomyopathy) caused by transthyretin mediated amyloidosis (ATTR-CM) in adults. These are the first FDA-approved treatments for ATTR-CM. Vyndaqel and Vyndamax have the same active… Read More »

Protein protects against non-alcoholic fatty liver disease

Researchers propose a possible therapeutic target to treat fatty liver, a disease for which there is currently no treatment. Non-alcoholic fatty liver disease describes several liver dysfunctions of varying severity, characterised by the accumulation of fat in hepatic cells and not caused by high alcohol consumption. This disease, one of the most common in developed… Read More »

HIV used to cure ‘bubble boy’ disease

US scientists say they used HIV to make a gene therapy that cured eight infants of severe combined immunodeficiency, or “bubble boy” disease. Results of the research, developed at a Tennessee hospital, were published in the New England Journal of Medicine. The babies, born with little to no immune protection, now have fully functional immune… Read More »