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The Orphan Disease Center, in collaboration with the Bow Foundation, is seeking grant applications that aim to further progress our understanding of the disease, the available therapeutic options, and investigating strategies to establish outcome measurements. The RFA could focus on one, or several, of the following aims to further advance GNAO1 research and therapeutic approaches:

• Novel therapeutic approaches, including, but not limited to, techniques in genome editing, RNAbased mechanisms, biologics, novel cell-based therapeutics, and development of novel therapeutic compounds, including through small molecule repurposing or screening against validated phenotypes in human cellular systems.

• Identification of short-term biomarkers that can monitor disease activity and treatment response.

• Establishment of outcome measures for future clinical trials.

• Development of therapeutic approaches in early symptomatic patients. • Supporting pilot clinical trials, preclinical trials, or animal model trials that promote drug repurposing strategies.

• Development of a standardized evaluation criteria for clinical projects allowing uniformity of patients as well as the severity and progression of the disease.

• Development of cellular models (i.e. oligodendrocytes) for evaluation of therapeutic options to translate for clinical use.