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Pacritinib

Pacritinib is an inhibitor of both wild-type and mutant (V617F) JAK2, as well as FMS-like tyrosine kinase 3 (FLT3), which was granted accelerated approval by the FDA in February 2022 for the treatment … [L40754] It provides a treatment option for patients who have MF with severe thrombocytopenia, which occurs in approximately one-third of MF patients and carries with it a particularly poor prognosis.

Vipadenant

Berberine

An alkaloid from Hydrastis canadensis L., Berberidaceae. It is also found in many other plants. … It is relatively toxic parenterally, but has been used orally for various parasitic and fungal infections and as antidiarrheal.

Minaprine

Minaprine is an amino-phenylpyridazine antidepressant reported to be relatively free of cardiotoxicity, drowsiness, and weight gain. … Minaprine is a psychotropic drug which has proved to be effective in the treatment of various depressive states. Like most antidepressants minaprine antagonizes behavioral despair.

Atidarsagene autotemcel

a lentiviral vector encoding the human arylsulfatase A (ARSA) gene. … [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

Teicoplanin

Teicoplanin is a glycopeptide antibiotic consisting of a mixture of several compounds, five major (named teicoplanin A2-1 through A2-5) and four minor (named teicoplanin RS-1 through RS-4). … All teicoplanins share a same glycopeptide core, teicoplanin A3-1, but differ in the length and conformation of side chains attached to their β-D-glucosamine moiety.

Dexrazoxane

It appears to inhibit formation of a toxic iron-anthracycline complex. … [PubChem] The Food and Drug Administration has designated dexrazoxane as an orphan drug for use in the prevention or reduction in the incidence and severity of anthracycline-induced cardiomyopathy.

ISS-1018

ISS 1018 is currently being investigated in combination with HBsAg vaccine as a prophylactic treatment for to prevent Hepatitis B in adults. … ISS 1018 is a short, synthetic, unmethylated CpG oligodeoxynucleotide (CpG ODN) with immunostimulatory activity.

Ustekinumab

[A187349] It was generated via recombinant human IL-12 immunization of human Ig (hu-Ig) transgenic mice. … Ustekinumab is a human immunoglobulin (Ig) G1 kappa monoclonal antibody directed against interleukin(IL)-12 and IL-23, which are cytokines that are involved in immune and inflammatory responses.

Edaravone

Edaravone is a free radical scavenger and neuroprotective agent with antioxidant properties.[A254257] It has three tautomers. … [L44002,L44012] Edaravone was also investigated in other disorders, such as Alzheimer's disease,[A19138] neuropathic pain, and ischemia-induced nerve injury.[A19139]