by Madaline Spencer | Mar 11, 2024
Alaa Hamed, MD, Global Head of Medical Affairs Rare Diseases at Sanofi, discusses the importance of rare disease registries. One of the fundamental challenges of rare diseases is the scarcity of data. While traditional medical research utilizes large...
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...
