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Zilganersen

[L64096] Alexander disease is caused by variants in the *GFAP* gene that lead to overproduction and toxic accumulation of GFAP in astrocytes, and dysfunction in astrocytes can over time damage neurons … [L64096] The FDA approved zilganersen on September 3, 2026 for the treatment of Alexander disease in pediatric and adult patients, making it the first and only disease-modifying treatment for the condition

Trofinetide

[A258453] Trofinetide is marketed as DAYBUE and DAYBUE STIX in the United States, as DAYBUE in Canada, and as DAYBU in the European Union. … Trofinetide is a novel synthetic analog of [glypromate], also known as glycine–proline–glutamate (GPE), a naturally occurring protein in the brain and the N-terminal tripeptide of insulin-like growth factor

Metyrapone

An inhibitor of the enzyme steroid 11-beta-monooxygenase. It is used as a test of the feedback hypothalamic-pituitary mechanism in the diagnosis of cushing syndrome.

Metronidazole benzoate

Trichlormethiazide

(From Martindale, The Extra Pharmacopoeia, 30th ed, p830)

Nirmatrelvir

Commission on January 27, 2022. … [L39544] It was fully approved by the FDA on May 25, 2023.

Lurasidone

Food and Drug Administration (FDA) for treatment of schizophrenia on October 29, 2010 and is currently pending approval for the treatment of bipolar disorder in the United States. … Lurasidone is an atypical antipsychotic developed by Dainippon Sumitomo Pharma. It was approved by the U.S.

Ferric maltol

[A189291] Ferric maltol was initially granted FDA approval on July 25, 2019, for use in adults. … Ferric maltol is an iron(III) atom complexed with 3 maltol molecules to increase the bioavailability compared to iron(II), without depositing it in the duodenum as insoluble ferric hydroxide and phosphate

Etranacogene dezaparvovec

Hemophilia B - also called factor IX deficiency or Christmas disease - is an X-linked genetic disorder resulting in an absence or deficiency of clotting factor IX. … [L44156] It delivers a copy of the deficient gene that results in cell transduction and an eventual increase in circulating factor IX activity.

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