Pharmaceutical preparations for diagnosis, prevention and treatment of diseases and disorders by gene therapy, namely, Friedreich's ataxia and congenital birth defects; Pharmaceuticals 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 the treatment of neurological, central nervous system, psychiatric and movement diseases, disorders and conditions; pharmaceutical preparations for the treatment of endocrine diseases, disorders and conditions; pharmaceutical preparations for the treatment of hormonal diseases, disorders and conditions; pharmaceutical preparations for the treatment of genetic diseases, disorders and conditions

'adren':46 'ataxia':18 'birth':21 'central':55 'condit':34,64,75,86,97 'congenit':20,45 'defect':22 'development':40 'diagnosi':4 'diseas':9,31,61,72,83,94 'disord':11,32,62,73,84,95 'encephalopathi':43 'endocrin':71 'epilepsi':38 'epilept':42 'friedreich':16 'gene':13 'genet':93 'hormon':82 'hyperplasia':47 'movement':60 'name':15,35 'nervous':56 'neurolog':54 'orphan':30 'pediatr':37 'pharmaceut':1,23,48,65,76,87 'prepar':2,49,66,77,88 'prevent':5 'psychiatr':58 'rare':28,36 'scn8a':39 'system':57 'therapi':14 'treatment':7,26,52,69,80,91

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