by Madaline Spencer and Scott Harwood | Mar 5, 2026
Bob Stevens, Group CEO of the MPS Society and Rare Disease Research Partners in the UK, discusses global efforts towards developing treatment options for patients with rare lysosomal storage disorders (LSDs). Mr. Stevens has been involved with the UK MPS...
by Madaline Spencer and Scott Harwood | Mar 4, 2026
Dawn Laney, MS, Genetic Counselor at Emory University School of Medicine, discusses the importance of newborn screening and the need for more effective treatments in infantile Krabbe disease. Krabbe disease is a lysosomal disorder that affects the entire body,...
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...
