Based on robust preliminary work, these companies are developing phase II and phase III clinical trials, essentially testing the safety and efficacy trends of therapies designed to target the mutant huntingtin and stop the disease process, but also aimed at controlling HD symptoms, such as motor and cognitive changes.
Many of these new studies will involve younger people in prodromal and very early disease stages, which is an important step in HD research. Additionally, given the disease complexity, companies such as Roche are pursuing multiple therapeutic approaches in parallel – besides Tominersen, an oral splicing modifier and an allele-specific ASO will soon be included in their preclinical study pipeline.
The times are changing for the HD community, with the growing number of important companies diligently working and showing that they are highly committed to change the lives of everyone affected by this disease. This is very good news and we wanted to share it with everyone!
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