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  2. Casimersen - Wikipedia

    en.wikipedia.org/wiki/Casimersen

    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]

  3. Tovorafenib - Wikipedia

    en.wikipedia.org/wiki/Tovorafenib

    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]

  4. Fenfluramine - Wikipedia

    en.wikipedia.org/wiki/Fenfluramine

    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. Disruptive mood dysregulation disorder - Wikipedia

    en.wikipedia.org/wiki/Disruptive_mood...

    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.

  6. List of gene therapies - Wikipedia

    en.wikipedia.org/wiki/List_of_gene_therapies

    Strimvelis: treatment for adenosine deaminase deficiency (ADA-SCID) Talimogene laherparepvec (Imlygic): treatment for melanoma in patients who have recurring skin lesions [17] Tisagenlecleucel (Kymriah): treatment for B cell lymphoblastic leukemia [18] Valoctocogene roxaparvovec (Roctavian): treatment for hemophilia A [19] [20] [21]

  7. 19 drug approvals in 2024 that had 'big clinical impact ... - AOL

    www.aol.com/19-drug-approvals-2024-had-093022917...

    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."

  8. How a new FDA-approved drug can — and can’t — help people ...

    www.aol.com/fda-approved-drug-t-help-121600044.html

    Just because the FDA approves a drug doesn’t necessarily mean that Medicare will pay for it for all patients. Before Leqembi was approved, another similar medication was approved called Aduhelm ...

  9. Pretomanid - Wikipedia

    en.wikipedia.org/wiki/Pretomanid

    It is in the nitroimidazole class of medications. [6] Pretomanid was approved for medical use in the United States in August 2019, [4] [7] and in the European Union in July 2020. [2] Pretomanid was developed by TB Alliance. [8] [4] [9] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [10]

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