One-Time Gene Therapy Fast Tracked for Duchenne Muscular Dystrophy
RGX-202 is an investigational one-time gene therapy designed to deliver a transgene for a novel microdystrophin.
RGX-202 is an investigational one-time gene therapy designed to deliver a transgene for a novel microdystrophin.
Olezarsen is an investigational LICA antisense medication designed to inhibit the production of apoC-III for patients at risk of disease due to elevated triglyceride levels.
Skyclarys activates the Nrf2 pathway which is involved in the cellular response to oxidative stress.
Previously, the treatment had been approved for patients 1 year of age and older.
The sNDA is supported by data from the phase 3, placebo-controlled APOLLO-B study.
CHAPLE disease is a rare genetic disorder caused by mutations in the CD55 gene.
Velmanase alfa is a recombinant form of human lysosomal alpha-mannosidase intended to provide or supplement natural alpha-mannosidase.
AT-GAA consists of cipaglucosidase alfa administered in conjunction with miglustat.
Bitopertin is an investigational oral, selective inhibitor of glycine transporter 1 designed to modulate heme biosynthesis by limiting glycine uptake.
Pegunigalsidase alfa is a long-acting recombinant, PEGylated, cross-linked α-galactosidase-A investigational product candidate.