Denali MPS IIIA Clinical Study

Denali MPS IIIA Clinical Study

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...
Clinical Trial RGX-111 for MPS I Halted

Clinical Trial RGX-111 for MPS I Halted

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...
Post-Trial Access TAK-609

Post-Trial Access TAK-609

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....