Pharmaceutical preparations for human use for the treatment of endocrine, hormonal, and genetic diseases, disorders, and conditions; Pharmaceutical preparations for human use for the prevention and treatment of diseases, disorders, and conditions by gene therapy; Pharmaceutical preparations for human use for the prevention and treatment of diseases, disorders, and conditions by gene therapy, namely, Friedreich's ataxia, Parkinson's disease, GBA1-mediated diseases, and congenital birth defects; Pharmaceutical preparations for human use for the treatment of rare and orphan diseases, disorders, and conditions, namely, rare pediatric epilepsies, SCN8A developmental and epileptic encephalopathy, and congenital adrenal hyperplasia; Pharmaceutical preparations for human use for the treatment of Huntington's Disease, chorea associated with Huntington's Disease, schizophrenia, adjunctive treatment of schizophrenia, cognitive impairment associated with schizophrenia, epilepsy, epileptic encephalopathy with continuous spike-and-wave during sleep, SCN8A-related epilepsy with encephalopathy, focal onset seizures, Parkinson's Disease, adjunctive treatment of Parkinson's Disease, Major Depressive Disorder, anhedonia in Major Depressive Disorder, inadequate response to treatment in Major Depressive Disorder, dyskinetic cerebral palsy, and neuro-immunological and neuroendocrinological diseases, disorders, and conditions

Providing medical information; Providing medical information in the field of endocrine, hormonal, and genetic diseases, disorders, and conditions; Providing medical information in the field of gene therapy; providing medical information in the field of psychiatric, neurological, central nervous system, and movement diseases, disorders, and conditions; Providing medical information in the field of Huntington's Disease and chorea associated with Huntington's Disease