Abstract: The disclosure provides for methods of predicting disease progression comprising detecting a modification of one or more disease markers in a glial cell or neuronal cell generated from a skin cell of the subject. The disclosed methods include methods of identifying subjects that are responsive to a therapeutic agent and methods of determining effectiveness of a therapeutic agent.

Abstract: Certain embodiments of the invention provide EphA4 targeting compounds and compositions comprising a compound described herein. Certain embodiments of the invention also provide methods of treating a neurological disease (e.g., Amyotrophic Lateral Sclerosis, Alzheimer's Disease) or cancer.

Abstract: Provided herein are methods of treating a subject comprising a SCN2A mutation, a mutated SCN2A voltage-gated sodium channel protein, a SLC6A1 mutation, a SCN1A mutation or an IRF2BPL mutation. Methods of treating a subject with elevated levels of basal mitochondrial respiration, mitochondrial ATP-linked respiration, or a combination thereof, are also provided. Methods of treating a subject with a seizure disorder and method of treating a subject with a neurodegenerative or neurological disorder with mitochondrial dysfunction, optionally, with elevated levels of basal and/or ATP-linked respiration, are provided. Methods of improving survival of motor neurons or other neuronal cells types, reducing mitochondrial basal and/or ATP-linked respiration, reducing cellular oxidative stress, or a combination thereof, additionally are provided. In exemplary embodiments, the method comprises administering to the subject copper-ATSM (CuATSM) in an effective amount.