Examining Tominersen as a Potential Treatment for Huntington Disease: The GENERATION HD2 trial
The phase 2 trial tests Roche’s investigational antisense oligonucleotide therapy tominersen in patients with early stages of Huntington disease, a disease with no FDA-approved therapies available.
Huntington disease (HD) is a rare, inherited neurological condition that progressively impacts movement, cognitive abilities, and behavior. Early signs can include subtle changes in mood, thinking, and behavior, which may not immediately interfere with daily functioning. As the disease advances, individuals may experience involuntary, jerky movements that interfere with basic tasks such as walking and eating, a stage known as early manifest HD. HD is caused by a mutation in the huntingtin gene (HTT), which leads to the production of a toxic form of the huntingtin protein (mHTT).