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Casimersen was approved for medical use in the United States in February 2021, [1] [2] [6] and it is the first FDA-approved targeted treatment for people who have a confirmed mutation of the DMD gene that is amenable to skipping exon 45. [2]
GoodRx released a list of 19 of the most influential drugs and vaccines approved by the FDA in 2024 to treat a variety of conditions. These medications are “slated to make a big clinical impact."
Disruptive mood dysregulation disorder (DMDD) is a mental disorder in children and adolescents characterized by a persistently irritable or angry mood and frequent temper outbursts that are disproportionate to the situation and significantly more severe than the typical reaction of same-aged peers.
Teriflunomide inhibits rapidly dividing cells, including activated T cells, which are thought to drive the disease process in MS. Teriflunomide may decrease the risk of infections compared to chemotherapy-like drugs because of its more-limited effects on the immune system.
Fenfluramine, sold under the brand name Fintepla, is a serotonergic medication used for the treatment of seizures associated with Dravet syndrome and Lennox–Gastaut syndrome. [ 3 ] [ 7 ] [ 4 ] It was formerly used as an appetite suppressant in the treatment of obesity , but was discontinued for this use due to cardiovascular toxicity before ...
Tovorafenib, sold under the brand name Ojemda, is a medication used for the treatment of glioma. [1] [2] It is a kinase inhibitor.[1]The most common adverse reactions include rash, hair color changes, fatigue, viral infection, vomiting, headache, hemorrhage, pyrexia, dry skin, constipation, nausea, dermatitis acneiform, and upper respiratory tract infection. [3]
Velsipity was approved by the US Food and Drug Administration (FDA) in October 2023. [6] [17] [18]In December 2023, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Velsipity, intended for the treatment of ulcerative colitis. [8]
The weight loss drug Zepbound, generically known as tirzepatide, is also now an approved medication to treat obstructive sleep apnea, per a Food and Drug Administration Dec. 20 press release.
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