by Madaline Spencer | Mar 3, 2026
Karen Bean, Health Economist at Orchard Therapeutics, discusses healthcare resource use for patients with Mucopolysaccharidosis type III (MPS III). MPS III, also known as Sanfilippo syndrome, is a genetic disorder characterized by the inability to break...
by Madaline Spencer | Mar 2, 2026
Kelly Guillo, Board Member of the Prader-Willi Syndrome Association in Georgia, discusses Prader-Willi syndrome (PWS) from the perspective of a caregiver. PWS is a rare genetic condition that affects many parts of the body but its most dominant symptom...
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
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...
