“Our Voices” Podcast: Saving Ryan
Saving Ryan is the inspiring story by physician-scientist Dr. Emil Kakkis about his journey to develop a new, first-ever treatment for MPS.
Discontinued TAK-609 Clinical Trial for MPS II
The Canadian MPS Society met directly with Takeda Pharmaceuticals on May 11th, when Takeda shared with us that they will discontinue development of...
New Born Screening #NBS4MPS
The Canadian MPS Society has been strongly advocating to add MPS I-H (Hurler Syndrome) to all newborn screening panels throughout Canada. To date,...
Connect With Rare Disease Communities!
Ultragenyx has relaunched their patient support interactive website, UltraRareAdvocacy.com with a refreshed design and more ways to connect with...
NICE Recommends Vimizim For Treating MPS IVA Morquio
We are excited to hear that Elosulfase alfa - branded as Vimizin and made by BioMarin - has been recommended by NICE (National Institute for...
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...