by adminmps | Sep 6, 2022 | Clinical Trials, News, Research
In our March edition we reported that Abeona Therapeutics had decided to end enrollment for their clinical trial for MPS IIIA children. We are now happy to share that Ultragenyx will be taking over the clinical trials for AAV gene therapy ABO-102 (now UX111). “Based...
by adminmps | Sep 6, 2022 | Clinical Trials, News, Research
We are excited to share the news that REGENXBIO Inc. has announced its intention to file a Biologics License Application (BLA) in 2024 using the FDA’s accelerated approval pathway for the treatment of Hunter Syndrome, Mucopolysaccharidosis Type II (MPS II). The...
by adminmps | Sep 6, 2022 | News, Resources
Ever wondered… How does gene therapy work?Why does it work better for some conditions that others?What is the difference between in vivo and ex vivo gene therapy?What are the ways of receiving gene therapy?How safe is it and what are the dilemmas surrounding gene...
by adminmps | Sep 6, 2022 | News, Resources
Matthew Ellinwood, DMV, PhD, Chief Scientific Officer at the MPS Society USA, provides an overview of the mucopolysaccharidoses (MPSs) and mucolipidoses (MLs). As Dr. Ellinwood explains, most MPSs are rare genetic conditions that are often difficult to diagnose...
by adminmps | Sep 6, 2022 | Clinical Trials, News
Homology Medicines have received clearance from Health Canada to initiate the juMPStart trial for HMI-203, a one-time, in vivo gene therapy candidate for the treatment of adults with Hunter Syndrome (MPS II). If you are interested in knowing more, join Homology...
by adminmps | Aug 24, 2022 | News, Past Event
The 2022 Canadian MPS Society Annual General Meeting will be held on: Monday 26 September 2022 7:30 pm – 8:30 pm EST (4:30 pm – 5:30 pm PST). The meeting will be held online. Access details will be sent out in a separate email to...
