by Madaline Spencer | Mar 2, 2026
Ida Vanessa D. Schwartz, MD, PhD, HCPA, Professor of Genetics at the Federal University of Rio Grande do Sul, discusses two-year follow up from GALILEO-1 of FLT201 gene therapy in adults with Gaucher disease type 1 (GD1). Gaucher disease refers to a...
by Madaline Spencer | Feb 27, 2026
The US Food and Drug Administration (FDA) has approved Desmoda (desmopressin acetate) oral solution for the management of arginine vasopressin deficiency (AVP-D). AVP-D is a rare neuroendocrine disorder caused by the loss or impaired function of vasopressinergic...
by Madaline Spencer | Feb 27, 2026
Kim Stephens, DBA, Executive Director of the Muenzer MPS Research & Treatment Center, discusses a survey of clinical guidelines for mucopolysaccharidosis II (MPS 2; Hunter syndrome). MPS II is an inherited disorder of carbohydrate metabolism that...
by Madaline Spencer | Feb 26, 2026
The US Food and Drug Administration (FDA) has granted accelerated approval to Loargys (pegzilarginase-nbln) for the treatment of patients ages 2 years and older with arginase 1 deficiency (ARG1-D). ARG1-D is a rare genetic disorder characterized by complete or...
by Madaline Spencer | Feb 26, 2026
The US Food and Drug Administration (FDA) has approved Venclexta (venetoclax) in combination with acalabrutinib therapy for untreated adult patients with chronic lymphocytic leukemia (CLL). CLL is a rare cancer of the white blood cells likely caused by a combination...
