News

PTC518 lowers mutant HTT, slows disease progression: Interim data

An investigational oral therapy from PTC Therapeutics called PTC518 safely and effectively reduces levels of the mutant huntingtin protein (mHTT) in Huntington’s disease patients, while being linked to trends of slower disease progression. That’s according to one-year interim data from the ongoing Phase 2a PIVOT-HD clinical trial…

HDSA 2024: Huntington’s disease clinical trials enrolling in US

Clinical trials testing either tominersen, an experimental therapy designed to target the underlying cause of Huntington’s disease, or dalzanemdor, an oral candidate that aims to improve cognitive abilities, are currently enrolling Huntington’s patients in the U.S. Updates on the therapies’ clinical programs were provided by representatives of their…

HDSA 2024: Community urged to participate in observational studies

The Huntington’s disease community was encouraged to step up and get involved in observational studies to help scientists better understand the neurodegenerative disorder and accelerate the development of potential treatments. Participating in research “is the greatest thing that we can do to help expedite treatments for HD [Huntington’s disease],”…

AMT-130 is first Huntington’s therapy to receive RMAT status

The U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) status to AMT-130, an experimental gene therapy for Huntington’s disease, the first time a Huntington’s treatment candidate has received such a designation. The RMAT designation is given to therapies that seek to treat, modify,…