Cipaglucosidase Alfa, Miglustat Under Review for Pompe Disease
AT-GAA consists of cipaglucosidase alfa administered in conjunction with miglustat.
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.
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.
Gene Therapy Gets Priority Review for Duchenne Muscular Dystrophy
SRP-9001 is an investigational gene transfer therapy designed to deliver a shortened, functional component of dystrophin to muscle tissue.
Oxlumo Indication Expanded to Include Patients With Advanced PH1
The approval was based on data from the phase 3 ILLUMINATE-C study that included 21 dialysis-independent and -dependent patients with PH1.
Leniolisib Gets Priority Review for Activated Phosphoinositide 3-Kinase Delta Syndrome
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.
FDA to Review Velmanase Alfa for Alpha-Mannosidosis
Velmanase alfa is a recombinant form of human alpha-mannosidase intended to provide or supplement natural alpha-mannosidase.
Orkambi Indication Expanded to Include More Children With Cystic Fibrosis
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.
Oral Glycogen Synthase Inhibitor Designated Orphan Drug for Pompe Disease
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.
- Loading...
Loading...
Next post in Medicine
Loading... 
Loading...