by Madaline Spencer | Mar 4, 2026
The US Food and Drug Administration (FDA) has granted accelerated approval of Yuviwel (navepegritide; previously TransCon CNP) for the treatment of patients two years of age and older with achondroplasia. Achondroplasia is a disorder of bone growth that prevents the...
by Madaline Spencer | Mar 3, 2026
The US Food and Drug Administration (FDA) has approved the supplemental Biologics License Application for Palynziq (pegvaliase-pqpz) to include pediatric patients ages 12 years and older with phenylketonuria (PKU). Phenylketonuria (PKU) is a genetic metabolic disorder...
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...
