Huntington’s disease (HD) is a rare progressive neurological disorder characterized by movement disorders, cognitive decline, behavioral changes, and psychiatric disturbances. Current treatment is entirely dependent on symptomatic therapies and includes a cocktail of antidepressants, antipsychotics, and antiepileptics to control patients’ cognitive, behavioral, and neurological symptoms. Treatment for chorea is limited to tetrabenazine and Teva’s Austedo (deutetrabenazine), which launched in 2017 in the United States. HD is a debilitating condition, and available treatments are only marginally effective, leaving tremendous need for effective disease-modifying therapies (DMTs). Ionis/Roche’s HTTRx/RG6042 is an antisense oligonucleotide being evaluated in Phase III trials for HD. The therapy has the potential to modify disease course and, accordingly, has garnered interest among treating physicians. Furthermore, the Phase II HD pipeline also includes a variety of additional symptom-targeting and potential disease-modifying therapies, any of which stands to capitalize on the significant commercial opportunity in HD if its efficacy is demonstrated.
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