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Keep up with what’s happening in the MPS community, industry and research.
News Feed
News from the MPS Community, industry and research.
To view posts by category, click on your chosen category which appears after the date in any of the posts.
Recruiting for Clinical Trial: MPS-II (Hunter Syndrome) with Pabinafusp-Alfa
Mathias Schmidt, PhD, President and CEO of JCR Pharmaceuticals USA, discusses long-term efficacy and safety data of pabinafusp-alfa (Izcargo) in...
Current Treatment Options Explained for Hurler Syndrome
(MPS I) Raymond Wang, MD, Metabolic Specialist and Director of the Multidisciplinary Lysosomal Storage Disorder Program at Children’s Hospital of...
Advocacy Groups Unveil ‘Roadmap’ for Sanfilippo Research, Care
A coalition of patient advocacy organizations has published a Global Roadmap for Sanfilippo Syndrome Therapies, in an effort to accelerate research...
Rapid Test Expected to Help in Developing Treatments for Sanfilippo
A new study shows a 30-minute, inexpensive, and easy-to-use test may help measure the activity of NDST1, one of the main enzymes involved in...
Abeona Ends Enrollment For MPS IIIA Clinical Trial
We are disappointed to hear that Abeona Therapeutics has decided to end enrollment for their clinical trial (Study ABT-003) for MPS IIIA children...
Resources for Courageous Parents
Courageous Parents Network is a non-profit organization and educational platform that orients, empowers and accompanies families and providers...
Research Highlights from the 2022 WORLD Symposium:
MPS I (Hurler syndrome) Orchard Therapeutics Announces Interim Data for OTL-203 Showing Positive Clinical Results in Multiple Disease Manifestations...
The Importance of Newborn Screening
Read Gianna’s story and how it changed the course of her life. Newborn screening gave Gianna a head start in her fight against MPS I. Today marks...