Compositions and methods for treating neurodegenerative, neurodevelopmental, myodegenerative, and lysosomal storage disorders

Abstract

Disclosed herein are compounds of Formula I wherein R¹ is H, NR⁸R⁹, CR⁸R⁹R¹⁰, or OR⁸; R⁸, R⁹ and R¹⁰ are each independently H, OH, substituted or unsubstituted aryl, or substituted or unsubstituted alkyl, cycloalkyl, heteroalkyl, cycloheteroalkyl, or heteroaryl; R², R⁴, R⁵, R⁶, and R⁷ are each independently H, OH, halogen, C₁₋₆ alkyl, or C₁₋₆ alkoxy; R³ is NR¹¹R¹² or OR¹¹; and R¹¹ and R¹² are each independently H, OH, substituted or unsubstituted aryl, or substituted or unsubstituted alkyl, cycloalkyl, heteroalkyl, cycloheteroalkyl, or heteroaryl. Isomers and pharmaceutically acceptable salts thereof are also disclosed. The described compounds are useful for treating or preventing a neurodegenerative disease, a neurodevelopmental disorder (e.g., attention deficit hyperactivity disorder, autism spectrum disorder), a myodegenerative disease, a prion disease, a lysosomal storage disease or cancer in a subject, e.g., a pediatric subject. [Image available in the PDF document]