FDA Places Clinical Hold on Investigational Gene Therapy for Phenylketonuria
BMN 307 is an investigational AAV5-phenylalanine hydroxylase gene therapy designed to normalize phenylalanine concentration levels.
Inborn Errors Of Metabolism
Mitapivat Granted Priority Review for Pyruvate Kinase Deficiency
Mitapivat is a first-in-class, oral, selective small molecule allosteric activator of pyruvate kinase-R enzymes.
Oral Myosin Inhibitor Fast Tracked for Becker Muscular Dystrophy
EDG-5506 is a selective, small molecule myosin inhibitor designed to protect injury-susceptible fast skeletal muscle fibers.
Novel Gene Therapy for Wilson Disease Gets Fast Track Status
VTX-801 is an investigational, novel rAAV-based gene therapy that delivers a shortened version of the ATP7B transgene.
Nexviazyme Approved for Late-Onset Pompe Disease
The Food and Drug Administration (FDA) has approved Nexviazyme (avalglucosidase alfa-ngpt), a hydrolytic lysosomal glycogen-specific enzyme, for the treatment of late-onset Pompe disease in patients 1 year of age and older. Pompe disease is a rare, degenerative muscle disorder caused by mutations in the acid alpha-glucosidase (GAA) gene, which leads to the abnormal buildup of…
Tesomet Receives Orphan Drug Status for Hypothalamic Obesity
Tesomet is an investigational fixed-dose combination therapy of tesofensine, a triple monoamine reuptake inhibitor, and metoprolol, a beta-1 selective blocker.
Bylvay Approved for Progressive Familial Intrahepatic Cholestasis
Bylvay is an oral reversible inhibitor of the ileal bile acid transporter.
Gene Therapy Candidate Fast Tracked for GM1 Gangliosidosis
LYS-GM101 is a gene therapy designed to replace the gene mutation and restore the production of the beta-galactosidase enzyme.
FDA to Review Vutrisiran for Polyneuropathy of Hereditary ATTR Amyloidosis
Vutrisiran is an investigational, subcutaneously-administered RNAi therapeutic designed to block the production of wild-type and mutant transthyretin protein.
FDA: More Data Needed to Support Arimoclomol in Niemann-Pick Disease Type C
Arimoclomol is an investigational oral drug candidate that is designed to amplify and sustain the cellular production of heat shock proteins.
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