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The purposes of this study are to identify persons with rapid-onset dystonia-parkinsonism (RDP), alternating hemiplegia of childhood (AHC), or other diseases caused by mutations of the ATP1A3 gene, document prevalence of the disease, and map its natural history. This study, which is a continuation of an earlier study begun by Dr. Allison Brashear, aims to more clearly identify the characteristics associated with ATP1A3 disease and to explore whether mutations in the gene are associated with atypical dystonias, Parkinson's disease, and other movement disorders. The study involves in-person or remote (telemedicine) neurological assessments and blood samples for genetic analysis.
A randomized, double-blind, placebo-controlled, Phase 2 study to assess the efficacy, safety, and pharmacokinetics of FNP-223 (oral formulation) to slow the disease progression of progressive supranuclear palsy (PSP)
This Phase 2 study is intended to help better understand the potential impact of SAGE-718 on the day-to-day functioning of participants with HD. Like the Dimension study, you may be eligible to participate in this trial if you are between 25 and 65 years of age and have genetically confirmed HD with pre-manifest to early-manifest presentation. Additionally, this study invites healthy volunteers to participate
This phase 2 study seeks to evaluate the effect of the study drug SAGE-718 on the cognition of participants with premanifest or recent HD diagnosis. You can participate if you are between 25 to 65 years of age and have been diagnosed with early HD
The goal of this phase 2 study is to see whether the study drug might delay PD by modifying the activity of a gene involved in PD called GBA1. You may be eligible to participate if you are between the ages of 30 and 80 years old, have had between 1 and 7 years of Parkinson’s disease symptoms, are able to walk without assistance, and are taking medication for Parkinson’s disease.
This study will test a new investigational cellular therapy product called Bemdaneprocel (stem cell derived neuronal cells) for people with Parkinson’s Disease who have 2.5 hours or more of daily OFF time. Patients are randomized to either stem cell therapy or placebo. The goal is to improve long-term PD management by improving their ability to move, slowing the disease's progress, and enhancing their quality of life. It also seeks to do this with fewer side effects than current treatments.