Leniolisib is a small molecule inhibitor of the delta isoform of the 110 kDa catalytic subunit of class IA PI3K with immunomodulating and potentially antineoplastic activities.
Velmanase alfa is a recombinant form of human alpha-mannosidase intended to provide or supplement natural alpha-mannosidase.
The safety profile of Orkambi was found to be similar in patients aged 1 to less than 2 years compared with those 2 years of age and older.
Pompe disease is a rare, degenerative muscle disorder caused by mutations in the acid alpha-glucosidase gene, which leads to the abnormal buildup of glycogen.
Xenpozyme is a hydrolytic lysosomal sphingomyelin-specific enzyme replacement therapy designed to replace deficient or defective ASM.
The APOLLO-B study included patients 18 years of age and older with ATTR amyloidosis (hereditary or wild-type) with cardiomyopathy.
Eplontersen is an investigational ligand-conjugated antisense medicine designed to reduce the production of TTR protein to treat both hereditary and nonhereditary forms of ATTR.
Imcivree is a melanocortin 4 receptor (MC4R) agonist that re-establishes MC4 receptor pathway activity to reduce hunger and promote weight loss.
Vutrisiran is an investigational, subcutaneously-administered RNAi therapeutic designed to block the production of wild-type and mutant transthyretin protein.
Omaveloxolone is an investigational Nrf2 activator.
