FDA approves rare liver disease drugErin Bastick
Dec. 17, 2024 – The FDA hasapprovedcrinecerfont, a first-in-class oral drug for treating classic congenital adrenal hyperplasia (CAH), a rare genetic condition present from birth. It is intended for patients ages 4 and older and should be used along with steroid medicines. Classic CAH is ...
The Food and Drug Administration has approved the first drug to treat an unusual condition that causes painful, curved erections in men. The agency says it approved the biotech drug Xiaflex (ZEE'-a-fleks) to treat Peyronie's (PEHR'-oh-nees) disease, which causes an abnormal bend in the ...
WASHINGTON, Aug. 8 (Xinhua) -- The United States Food and Drug Administration (FDA) approved on Wednesday a new drug for two rare, hard-to-treat types of non-Hodgkin lymphoma. FDA approved Poteligeo injection for intravenous use for the treatment of adult patients with relapsed or refractory...
The new drug,mitapivat(Pyrukynd,from Agios Inc),was approved on the basis of clinical trials that showed that it significantly improved hemolysis andanemiain patients with PK deficiency. PK deficiency is rare. In clinical practice, its frequency is approximately three to nine cases per one mill...
The FDA granted this drug orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. 美国食品和药物管理局批准了这种药物孤儿药物名称,此举为协助和鼓励开发稀有疾病药物提供了激...
The FDA approved IntraBio Inc.'s Aqneursa (levacetylleucine) yesterday for the treatment of ultra rare neurodegenerative disease Niemann-Pick Disease, Type C (NPC), according to a news release. This makes Aqneursa the second NPC drug approved within a week and the first standalone FDA-ap...
[2] FDA approves new treatment from Biogen for rare form of ALS. Retrieved April 27, 2023 from网页链接 [3] FDA approves Biogen’s ALS drug tofersen, paving new road for accelerated pathway in neuro diseases. Retrieved April 27, 2023 from网页链接 ...
syndrome,” said Ann Farrell, M.D., director of the Division of Nonmalignant Hematology in the FDA’s Center for Drug Evaluation and Research. “FDA is committed to helping develop safe and effective treatment options for this group of rare and debilitating blood diseases ...
The very rare, inherited blood clotting disorder called cTTP is caused by a disease-causing mutation in the ADAMTS13 gene, which is responsible for making an enzyme, also named ADAMTS13, that regulates blood clotting. A deficiency in this enzyme causes blood clots to form i...