by Madaline Spencer | Mar 8, 2024
Petra Oliva, PhD, Archimed Life Science, discusses alpha mannosidosis in patients with mucopolysaccharidosis (MPS)-like phenotypes. Alpha mannosidosis is a lysosomal storage disorder, characterized by intellectual disability, hearing loss, ataxia,...
by Madaline Spencer | Mar 7, 2024
Barbara Burton, MD, Clinical and Biochemical Geneticist at Northwestern University and Ann & Robert H. Lurie Children’s Hospital of Chicago, discusses enzyme replacement therapy in patients with mucopolysaccharidosis (MPS) IVA. Mucopolysaccharidoses...
by Madaline Spencer | Mar 6, 2024
Mathias Schmidt, PhD, President and Chief Executive Officer of JCR USA, describes their current clinical trials for patients with mucopolysaccharidosis (MPS) I and II. Mucopolysaccharidoses (MPSs) are a group of rare genetic lysosomal storage diseases,...
by Madaline Spencer | Mar 5, 2024
In a significant development, Chiesi Global Rare Diseases has announced positive results from the Phase 3 BALANCE study, evaluating the efficacy, safety, and tolerability of pegunigalsidase alfa in adult patients with Fabry disease. Fabry disease is a...
by Madaline Spencer | Mar 4, 2024
Priya Kishnani, MD, Professor of Pediatrics at Duke University Medical Center, discusses recent clinical trials in Pompe disease. Pompe disease is a rare genetic, lysosomal disorder that causes progressive weakness in heart and skeletal muscles. It is...
