Clinical challenges and future therapeutic approaches for neuronal ceroid lipofuscinosis

Treatment of the neuronal ceroid lipofuscinoses, also known as Batten disease, is at the start of a new era because of diagnostic and therapeutic advances relevant to this group of inherited neurodegenerative and life-limiting disorders that affect children. Diagnosis has improved with the use of co...

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Veröffentlicht in:	Lancet neurology 2019-01, Vol.18 (1), p.107-116
Hauptverfasser:	Mole, Sara E, Anderson, Glenn, Band, Heather A, Berkovic, Samuel F, Cooper, Jonathan D, Kleine Holthaus, Sophia-Martha, McKay, Tristan R, Medina, Diego L, Rahim, Ahad A, Schulz, Angela, Smith, Alexander J
Format:	Artikel
Sprache:	eng
Schlagworte:	Age Ataxia Children Clinical trials Convulsions & seizures Dementia Deoxyribonucleic acid Diagnosis Disease Progression DNA Drugs Enzyme Replacement Therapy Enzymes Epilepsy FDA approval Genes Genetic Therapy - methods Genotype & phenotype Humans Molecular modelling Mutation Nervous system Neuronal ceroid lipofuscinosis Neuronal Ceroid-Lipofuscinoses - drug therapy Neuronal Ceroid-Lipofuscinoses - genetics Neuronal Ceroid-Lipofuscinoses - therapy Stem cells Transplants & implants
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