Ultragenyx has relaunched their patient support interactive website, UltraRareAdvocacy.com with a refreshed design and more ways to connect with rare disease communities. Visitors will find: More rare experiences from individuals and families living with...
We are excited to hear that Elosulfase alfa – branded as Vimizin and made by BioMarin – has been recommended by NICE (National Institute for Health and Care Excellence) for treating MPS IVA Morquio in England and Wales. Newly diagnosed patients and...
Mathias Schmidt, PhD, President and CEO of JCR Pharmaceuticals USA, discusses long-term efficacy and safety data of pabinafusp-alfa (Izcargo) in mucopolysaccharidosis type II (MPS II; Hunter syndrome). (Data from this study was recently presented...
(MPS I) Raymond Wang, MD, Metabolic Specialist and Director of the Multidisciplinary Lysosomal Storage Disorder Program at Children’s Hospital of Orange County, gives an overview of the treatment landscape for mucopolysaccharidosis type I. There are two MPS I...
A coalition of patient advocacy organizations has published a Global Roadmap for Sanfilippo Syndrome Therapies, in an effort to accelerate research and develop effective therapies and care for families affected by Sanfilippo syndrome. “The Roadmap aims to capture the...
