uniQure announces alignment with FDA on key elements of Accelerated Approval pathway for AMT-130 in Huntington’s disease 10 December 2024: – uniQure N.V., a gene therapy company advancing therapies for patients with severe medical needs, has announced that the company reached agreement with the U.S Food and Drug Administration (FDA) on key elements of an…
Category: Huntington’s disease
Cognitive impairment in HD
Phase 2 DIMENSION Study fails to meet primary endpoint Sage Therapeutics announced that its Phase 2 DIMENSION Study of dalzanemdor (SAGE-718) for cognitive impairment in Huntington’s disease failed to meet its primary and secondary endpoints. Dalzanemdor showed no statistically significant or clinically meaningful improvement versus placebo in the Symbol Digit Modalities Test at Day 84…
Phase 2 LUMINARY Study of SAGE-718 in Patients with Mild Cognitive Impairment and Mild Dementia due to Alzheimer’s Disease
Sage Therapeutics presents promising results from LUMINARY Study Data presented during the Emerging Science Session at the American Academy of Neurology’s Annual Meeting, April 2022 The LUMINARY Study is a Phase 2, open-label study evaluating the safety, tolerability and efficacy of SAGE-718 once daily in individuals with mild cognitive impairment and mild dementia due to…
PIVOT-HD Phase 2 Clinical Trial in Huntington’s Disease
PTC Therapeutics, Inc. announced on 31st March 2022 the initiation of the PIVOT-HD Phase 2 clinical trial evaluating PTC518 in people with Huntington’s disease (HD). PIVOT-HD is a global trial starting in the United States. PTC518 is an oral, small molecule splicing modifier that was specifically designed to selectively lower huntingtin mRNA and protein. There…
