Weight Loss Surgery May Prolong Life for People With Diabetes
Obesity in people with diabetes can shorten their lifespan. Researchers compared long-term survival outcomes with regard to use of traditional weight loss methods vs bariatric surgery.
Diagnosis & Disease Information
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.
The Potential Dangers of Ketogenic Diets in Kidney Disease
A recent review underscores the possible adverse effects of ketogenic diets, including increased risks for heart disease, cancer, diabetes, and kidney stones.
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.
Major Cardiovascular Events Linked to Metabolic Acidosis
Cardiovascular events are the leading cause of death among patients with chronic kidney disease, and metabolic acidosis in this population is underdiagnosed.
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.
Wegovy Approved for Chronic Weight Management
Wegovy contains semaglutide, a GLP-1 receptor agonist.
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