Arkuda Therapeutics is developing therapies for neurodegenerative diseases by targeting the root cause. Novel understanding of the cause of neurodegenerative diseases is attributed to lysosomal dysfunction that can lead to substrate and protein aggregation, inflammation and cellular damage. For example, some patients with frontotemporal dementia (FTD) possess mutations in a gene which codes for a lysosomal protein called granulin. Arkuda is discovering new insights into the importance of progranulin in regulating lysosomal health and using these insights to develop novel drugs to treat neurodegenerative disorders.
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