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Eplontersen

[A262975] Hereditary transthyretin-mediated amyloidosis is caused primarily by pathogenic variants of the TTR gene, leading to the formation and thus accumulation of insoluble and misfolded amyloid in … This approval was based on positive results demonstrated in the 35-week interim analysis from the Phase 3 NEURO-TTRansform study, where a consistent improvement in the Neuropathy Impairment Score +7 (mNIS

Thiamylal

(From Martindale, The Extra Pharmacopoeia, 30th ed, p919)

Aficamten

[L54783] Aficamten works by attenuating myocardial hypercontractility and left ventricular outflow tract obstruction, which are characteristics of oHCM.[A275293] … [L54788] Approved by the FDA on December 19, 2025, aficamten is indicated for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and

HSD-016

Hsd 016 is under investigation in clinical trial NCT00838461 (Study Evaluating the Safety, Pharmacokinetics (PK), and Pharmacodynamices (PD) of HSD-016).

scAAV9.U7.ACCA

scAAV9.U7.ACCA is a non-replicating recombinant adeno-associated virus vector containing 4 antisense sequences designed to target exon 2 in the human DMD gene under the control of U7 small nuclear non-coding … Developed by Nationwide Children’s Hospital, it is being investigated for the treatment of Duchenne muscular dystrophy.

Etranacogene dezaparvovec

[L45255] Etranacogene dezaparvovec was approved by the EMA in February 2023[L45439,L45444] and by Health Canada in October 2023.[L48801] … [L44156] It delivers a copy of the deficient gene that results in cell transduction and an eventual increase in circulating factor IX activity.

Desonide

A nonfluorinated corticosteroid anti-inflammatory agent used topically for dermatoses.

Hydrocortisone valerate

Tolrestat

It was sold under the tradename Alredase but was discontinued by Wyeth in 1997 because of the risk of severe liver toxicity and death. … While it was approved for marketed in several countries, it failed a Phase III trial in the U.S. due to toxicity and never received FDA approval.

Cyclandelate

It may cause gastrointestinal distress and tachycardia. Cyclandelate is not approved for use in the U.S. or Canada, but is approved in various European countries.