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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]
Fluvoxamine, sold under the brand name Luvox among others, is an antidepressant of the selective serotonin reuptake inhibitor (SSRI) class. [8] It is primarily used to treat major depressive disorder and, perhaps more-especially, obsessive–compulsive disorder (OCD), [9] but is also used to treat anxiety disorders [10] such as panic disorder, social anxiety disorder, and post-traumatic stress ...
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.
The one-time therapy, branded as Lenmeldy in the U.S., is approved for children in certain stages of disease progression, the Food and Drug Administration (FDA) said. US approves first gene ...
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 ...
5-Trifluoromethyl-2,4(1H,3H)-pyrimidinedione (FTY), the main metabolite. Pharmacokinetic data of oral trifluridine have only been evaluated in combination with tipiracil, which significantly affects biotransformation of the former. At least 57% of trifluridine are absorbed from the gut, and highest blood plasma concentrations are reached after ...
Lumateperone, sold under the brand name Caplyta, is an atypical antipsychotic medication of the butyrophenone class. It is approved for the treatment of schizophrenia as well as bipolar depression, as either monotherapy or adjunctive therapy (with lithium or valproate). [2] It is developed by Intra-Cellular Therapies, licensed from Bristol ...