Huntington's Disease Community Perspectives on Desired Characteristics of Disease Modifying Therapies.

ABSTRACT:

Background

Promising disease modifying therapies for Huntington's disease are now entering pivotal trials, raising questions of what patients and families consider successful outcomes. Consistent with an ongoing movement to incorporate patient preferences into the development of new therapies, we conducted a pilot study to assess Huntington's disease community views on emerging DMTs to assist in planning large-scale studies of patient preferences.

Methods

Semi-structured interviews were conducted with members of the Huntington's community (manifest disease, at-risk, and family/caregivers). Participants were asked which symptoms they believed should be targeted with novel treatments, as well as potential benefits and tradeoffs of delaying symptom onset versus prolonging late-

SUBMITTER: Gornick MC 

PROVIDER: S-EPMC7845464 | biostudies-literature | 2021 Jan

REPOSITORIES: biostudies-literature