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Tividenofusp alfa

Tividenofusp alfa is a first-in-class, brain-penetrant enzyme replacement therapy (ERT) indicated for the treatment of Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome . Hunter syndrome is a rare, X-linked lysosomal ...

Sapropterin

Sapropterin (tetrahydrobiopterin or BH4) is a cofactor in the synthesis of nitric oxide. It is also essential in the conversion of phenylalanine to tyrosine by the enzyme phenylalanine-4-hydroxylase; the conversion of tyrosine to L-dopa ...

Oteseconazole

Oteseconazole is an azole metalloenzyme inhibitor that targets fungal CYP51. CYP51, also known as 14α demethylase, participates in the formation of ergosterol, a compound that plays a vital role in the integrity of cell membranes. By bin...

Cytisine

Cytisine is an alkaloid naturally derived from the Fabaceae family of plants including the genera Laburnum and Cytisus.

Pomaglumetad methionil

LY2140023 is an investigational drug from Lilly, which is being developed as a new treatment option for schizophrenia.

IN01

Of note, the IN01 vaccine is being tested in a Phase I clinical trial in patients with fibrosing interstitial lung disease resulting from SARS-CoV-2 infection (NCT04537130).

Hydrocortisone aceponate

Palovarotene

[L39990,L40020] It has been granted rare pediatric disease and breakthrough therapy designations from the US FDA, although a previously submitted NDA was withdrawn in August 2021 pending the resubmission

Folitixorin calcium

ANX-510 (CoFactor) is a folate-based biomodulator drug being developed to enhance the activity and reduce associated toxicity of the widely used cancer chemotherapy, 5-fluorouracil (5-FU). CoFactor use with 5-FU is being evaluated in cli...

NasoVAX

The compound has already shown to be tolerated with no adverse effects or fever in healthy individuals with a nearly 6-fold cellular immune response in comparison to an injectable vaccine[L16313].