Alvelestat Designated Orphan Drug for Alpha-1 Antitrypsin Deficiency
Alvelestat is a novel, oral small molecule designed to inhibit neutrophil elastase.
Metabolic Disorders
Rethymic Approved for Pediatric Congenital Athymia
Rethymic is engineered human allogeneic thymus tissue designed to reconstitute the immune system in pediatric patients with congenital athymia.
Novel Sodium Phenylbutyrate Formulation Under Review for Urea Cycle Disorders
ACER-001, a nitrogen-binding agent, is a powder formulation of sodium phenylbutyrate designed using a microencapsulation process.
Cipaglucosidase Alfa, Miglustat Under Review for Pompe Disease
AT-GAA consists of cipaglucosidase alfa administered in conjunction with miglustat.
Depression, Anxiety Associated with Osteocalcin, Cortisol in Hyperparathyroidism
Anxiety and depression in people with hyperparathyroidism may be related to levels of 2 key hormones in the body, according to researchers.
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.
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…
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