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Nusinersen

An antisense oligonucleotide that induces survival motor neuron (SMN) protein expression, it was approved by the U.S. … FDA in December, 2016 as Spinraza for the treatment of children and adults with spinal muscular atrophy (SMA). It is adminstrated as direct intrathecal injection.

Rezafungin

[L45633,L45658] It was subsequently approved in the EU in November of the same year. [L52745] … Rezafungin is an echinocandin antifungal drug.

Ferric chloride

Sertindole

It was first marketed in 1996 in several European countries before being withdrawn two years later because of numerous cardiac adverse effects. … It is a phenylindole derivative used in the treatment of schizophrenia.

Eltrombopag

ITP is a condition that may cause unusual bruising or bleeding due to an abnormally low number of platelets in the blood. … Eltrombopag is used to treat low blood platelet counts in adults with chronic immune (idiopathic) thrombocytopenia (ITP), when certain other medicines, or surgery to remove the spleen, have not worked

gamma-Hydroxybutyric acid

It is currently regulated in the US and sold by Jazz Pharmaceuticals under the name Xyrem. However, it is important to note that GHB is a designated Orphan drug (in 1985). … GHB is a naturally occurring substance found in the central nervous system, wine, beef, small citrus fruits and almost all other living creatures in small amounts.

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]

Sulbactam

[L50131] When given in combination with β-lactam antibiotics, sulbactam produces a synergistic effect as it blocks the enzyme responsible for drug resistance by hydrolyzing β-lactams.[A263296]

Moroctocog alfa

extended half-life compared with conventional factor VIII due to conjugation to the dimeric Fc domain of human immunoglobulin G1, a long-lived plasma protein [A31551]. … Moroctocog alfa is approved by Health Canada and by the European Medicines Agency for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in patients with hemophilia

Fludrocortisone

Fludrocortisone is a synthetic mineralocorticoid used in conjunction with [hydrocortisone] to replace missing endogenous corticosteroids in patients with adrenal insufficiency.