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Cedazuridine

[A215107, A215112, A215117, A215127, L14897] Cedazuridine was first reported in 2014,[A215107] and was subsequently approved by the FDA on July 7, 2020, in combination with [decitabine] for sale by … Myelodysplastic syndromes (MDS) are a group of hematopoietic neoplasms that give rise to variable cytopenias progressing to secondary acute myeloid leukemia (sAML), which is invariably fatal if untreated

Pegvaliase

Phenylketonuria is a rare autosomal recessive disorder that is characterized by deficiency of the enzyme phenylalanine hydroxylase (PAH) [A33284] and affects about 1 in 10,000 to 15,000 people in the United … The presence of a PEG moiety in pegvaliase-pqpz allows a reduced immune response and improved pharmacodynamic stability [A33284].

Cannabidiol

In addition to the well-known activity on CB1 and CB2 receptors, there is further evidence that CBD also activates 5-HT1A/2A/3A serotonergic and TRPV1–2 vanilloid receptors, antagonizes alpha-1 adrenergic … changed significantly with regards to its usefulness in a number of medical conditions ranging from anxiety to epilepsy.

Metreleptin

Metreleptin, a recombinant analog of the human hormone leptin, is an orphan drug used to treat complications of leptin deficiency in people with lipodystrophy. … Metreleptin is produced in _E. coli_ and differs from native human leptin by the addition of a methionine residue at its amino terminus.

Ferrous ascorbate

Lusutrombopag

This hematological abnormality, especially in cases of severe thrombocytopenia (platelet count <50,000/μL), creates challenges to patients requiring invasive medical procedures where there is a significant … Lusutrombopag is currently in phase III development in various European countries including Austria, Belgium, Germany, and the UK [A36730].

Fencamfamin

Fencamfamin (Glucoenergan, Reactivan) is a stimulant which was developed in the 1960s as an appetite suppressant. … It is around half the potency of [dexamphetamine], and is prescribed at a dose of 10-60mg, although abusers of the drug tend to rapidly develop tolerance and escalate their dose.

Deutetrabenazine

This allows less frequent dosing and a lower daily dose with improvement in tolerability [A32043]. … It is a hexahydro-dimethoxybenzoquinolizine derivative and a deuterated [DB04844] [A32046].

Raxibacumab

Raxibacumab is produced by recombinant DNA technology in a murine cell expression system. FDA approved on December 14, 2012. … Raxibacumab is a human IgG1λ monoclonal antibody that binds the protective antigen (PA) component of B. anthracis toxin. Raxibacumab has a molecular weight of approximately 146 kilodaltons.

Levomilnacipran

Levomilnacipran is a selective serotonin and norepinephrine reuptake inhibitor (SNRI), although it is a more potent inhibitor of norepinephrine reuptake than serotonin reuptake. … [L47956] While levomilnacipran was previously investigated and proposed as a potential treatment for stroke in Europe, the EMA decided against this use.[L48011]