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Plecanatide

Plecanatide is a drug approved in January 2017 by the FDA for the treatment of chronic idiopathic constipation (CIC). … It should not be used in children less than six years of age, and should be avoided in patients six years to 18 years of age

Dronedarone

Atrial fibrillation is a common sustained arrhythmia where the treatment primarily focuses on stroke prevention and symptom management. … [A34604] Similar to [amiodarone], dronedarone is a multichannel blocker that works to control rhythm and rate in atrial fibrillation.

Fosdenopterin

In 2009 a recombinant, E. coli-produced cPMP was granted orphan drug designation by the FDA, becoming the first therapeutic option for patients with MoCD type A. … Molybdenum cofactor deficiency (MoCD) is an exceptionally rare autosomal recessive disorder resulting in a deficiency of three molybdenum-dependent enzymes: sulfite oxidase (SOX), xanthine dehydrogenase

Efanesoctocog alfa

episodes in patients with hemophilia A. … [A257464,A257469] In February 2023, efanesoctocog alfa was approved by the FDA as a new class of factor VIII therapy for hemophilia A.[L45379,L45414]

Glycopyrronium

[L33105] Glycopyrronium is commonly prescribed as a first line treatment for a wide variety indications and is considered to have a wider therapeutic window than [tiotropium]. … [A233535,A233540] Early research into glycopyrronium use was for its indication as an adjunct therapy in the treatment of peptic ulcers.

Silver citrate

Rezafungin

Rezafungin has a half-life higher than 130 hours and can be administered once a week instead of daily. … [L45633,L45658] It was subsequently approved in the EU in November of the same year. [L52745]

Eplontersen

Eplontersen is a transthyretin-directed antisense oligonucleotide with 3 covalently linked e N-acetyl galactosamine residues for hepatic delivery. … 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

Elexacaftor

[A187361] While dual corrector/potentiator combination therapy has proven useful in the treatment of a subset of CF patients,[A187358] their use is typically limited to patients who are homozygous for … [A187361] Elexacaftor, along with [VX-659], was designed to fill the need for an efficacious CF therapy for patients who are heterozygous for _F508del-CFTR_ and a gene that does not produce protein or

Atidarsagene autotemcel

[L45191] Libmeldy was granted orphan designation by the EMA in April 2007, and was issued a marketing authorization in the EU in December 2020 for the treatment of certain manifestations of metachromatic … a lentiviral vector encoding the human arylsulfatase A (ARSA) gene.