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Showing 3 matches for the following: Inventor: "Asokan, Aravind" ×
Gene therapy for ATP1A3-Related Neurologic Disorders
Unmet Need Alternating Hemiplegia of Childhood (AHC) is a rare neurologic disorder affecting children, causing episodes of temporary paralysis, dystonia, and severe behavioral issues, with prevalence estimates ranging from 1 in 1 million to 1…
Gene therapy for long-term treatment of GSDIa
Unmet Need Glycogen storage disease type Ia (GSDIa), or von Gierke disease, is a rare heritable metabolic disorder that impacts the liver and kidneys due to an inability to break down glycogen. An estimated 1…
Methods and compositions for treating protein aggregation diseases
Unmet Need Neurodegenerative disease affects more than 50 million people per year. Certain neurodegenerative diseases, like Alzheimer’s disease, result from the accumulation of amyloid-forming proteins in the brain, which results in cognitive impairment and has…