X-linked dystonia Parkinsonism (XDP) is a neurodegenerative disease that affects men whose mothers originate from the island of Panay, Philippines. Due to the relatively isolated region in which those with XDP live, and the rarity of the disease itself, limited data regarding the natural history is currently available. The goal of this application is to solidify an already established collaboration for a natural history study and the collection of DNA and other biological fluids. Natural history studies are important for understanding the etiology, range of manifestations and progression of a rare disease. Biospecimens are crucial to understand the underlying pathology at both the molecular and cellular level. In addition, investigators in the Philippines will undergo training in clinical research methodology in the United States. The training will improve their ability to conduct independent research on XDP in the Philippines.
X-linked dystonia Parkinsonism (XDP) is a neurodegenerative disease with features of parkinsonism and dystonia, and may be considered as one of the Parkinson's Plus Syndromes. Thus, data from a well-done natural history study of XDP may also assist in understanding the clinical course and subsequently affect drug development in the other rare Parkinson's Plus Syndromes.
