by adminmps | Dec 13, 2023 | Clinical Trials, News, Research
December 12, 2023 Dear Sanfilippo syndrome Type A Community, We are pleased to share that a Phase 1/2 clinical study of DNL126, an investigational enzyme replacement therapy designed to address the behavioral, cognitive, and physical symptoms in Sanfilippo syndrome...
by adminmps | Nov 9, 2023 | Clinical Trials, News
November 8, 2023 Dear MPS I community, This afternoon REGENXBIO Press Release 8Nov2023, we announced a corporate restructuring that impacts our development of potential AAV gene therapies for MPS I. We are disappointed to share that REGENXBIO will no longer be moving...
by adminmps | Apr 4, 2023 | Clinical Trials, News
Takeda has announced a Post-Trial Access program for patients currently enrolled in extension trials for TAK-609, a treatment for MPS II Hunter syndrome. This program will provide continued access for patients who have benefited from treatment on existing studies....
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 | 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...
