Dystonia consists of involuntary movements, abnormal posturing and pain. In adults, dystonia symptoms present in a particular region of the body and cause significant disability due to pain as well as impairment in activities of daily living and employment. The most common subtypes begin in adults, in a specific body region. Involvement of neck muscles (cervical dystonia) leads to abnormal head movements that make it difficult to hold the head still for basic activities such as reading, driving, or working on the computer. Involvement of the eye region (blepharospasm) is associated with spasms that prevent patients from seeing normally. Involvement of the larynx (laryngeal dystonia) with spasm of laryngeal muscles leads to difficulty speaking. The current gold standard treatment, botulinum toxin (BoNT), has limitations?painful, frequent injections due to ?wearing off? of treatment effect; expense; and expected side effects like swallowing difficulty and weakness. There is a clear therapeutic gap in our current treatment options for focal dystonia and also a clear need for an effective novel treatment. Testing any novel treatment is complicated because most adults with focal dystonia are treated with BoNT. This proposal will develop an app-based patient-reported outcome (PRO) measure to prepare for future clinical trials in the most common types of dystonia as noted above. This app-based PRO will measure changes in dystonia symptoms over a BoNT injection cycle to characterize individual temporal responses and to determine effect estimates for future trials. Finally, the PRO will be correlated with standard clinical rating scales currently in use. Successful completion of this project will lead to better clinical trial readiness in dystonia.
