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.
Diagnosis & Disease Information
Olezarsen Fast Tracked for Familial Chylomicronemia Syndrome
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 Approved for Friedreich Ataxia
Skyclarys activates the Nrf2 pathway which is involved in the cellular response to oxidative stress.
Livmarli Approval Expands to Infants as Young as 3 Months With Alagille Syndrome
Previously, the treatment had been approved for patients 1 year of age and older.
Patisiran Under Review for Cardiomyopathy of ATTR Amyloidosis
The sNDA is supported by data from the phase 3, placebo-controlled APOLLO-B study.
Pozelimab Gets Priority Review for CHAPLE, a Rare Hereditary Immune Disease
CHAPLE disease is a rare genetic disorder caused by mutations in the CD55 gene.
Lamzede Approved for Noncentral Nervous System Manifestations of Alpha-Mannosidosis
Velmanase alfa is a recombinant form of human lysosomal alpha-mannosidase intended to provide or supplement natural alpha-mannosidase.
Cipaglucosidase Alfa, Miglustat Under Review for Pompe Disease
AT-GAA consists of cipaglucosidase alfa administered in conjunction with miglustat.
Bitopertin Granted Orphan Drug Status for Erythropoietic Protoporphyria
Bitopertin is an investigational oral, selective inhibitor of glycine transporter 1 designed to modulate heme biosynthesis by limiting glycine uptake.
FDA Accepts Resubmitted BLA for Pegunigalsidase Alfa for Fabry Disease
Pegunigalsidase alfa is a long-acting recombinant, PEGylated, cross-linked α-galactosidase-A investigational product candidate.
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