If approved, UX111 will be the first approved therapy for the treatment of Sanfilippo syndrome Type A, a rare disease affecting young children ...

Denali Therapeutics solved a major challenge in Hunter syndrome treatment. Now it's ready to take its technology further.

Our bodies perform many critical metabolic reactions, using a wide range of enzymes and other molecules. When there is a problem with one of these metabolism-related molecules because of a genetic ...

A surprising new study reveals that blocking a supposedly protective enzyme, Caspase-2, could actually backfire—raising the risk of chronic liver damage and cancer over time. Researchers found that ...

Researchers at KAIMRC have established a link between a rare childhood disease and a newly identified gene mutation, as well as previously unknown complications. “This is the latest of more than 50 ...

Patients in the EU with rare disease thymidine kinase 2 deficiency (TK2d) have their first approved therapy, UCB's Kygevvi, ...

Researchers from Arizona, including Arizona State University and Mayo Clinic Arizona, set out to shed more light on the ...

Serum levels of B vitamins don't adequately reflect nutritional state for patients with chronic kidney disease (CKD).

The FDA has given accelerated approval to Avlayah as a treatment for neurologic manifestations of Hunter syndrome, also known ...

The FDA has approved Denali Therapeutics’ enzyme replacement therapy for a genetic lysosomal storage disease after a string ...

FitIndiaHitIndia: Know the best exercises to reduce fatty liver naturally, improve metabolism, support weight loss, and boost overall liver health with simple, effective daily routines.

Chronic pancreatitis is now recognized as a complex fibroinflammatory disorder involving genetic, environmental, and ...