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Neurology, ISSN 0028-3878, 03/2016, Volume 86, Issue 10, pp. 890 - 897
OBJECTIVE:To examine safety, tolerability, pharmacokinetics, and preliminary clinical efficacy of intrathecal nusinersen (previously ISIS-SMNRx), an antisense... 
PUNCTURE HEADACHE | ANTISENSE OLIGONUCLEOTIDE | FUNCTIONAL MOTOR SCALE | CLINICAL NEUROLOGY | Injections, Spinal | Muscular Atrophy, Spinal - diagnosis | Follow-Up Studies | Humans | Child, Preschool | Male | Dose-Response Relationship, Drug | Adolescent | Female | Muscular Atrophy, Spinal - drug therapy | Oligonucleotides - administration & dosage | Child | Cohort Studies | 324 | 117 | 227 | 252
Journal Article
Nature Neuroscience, ISSN 1097-6256, 03/2017, Volume 20, Issue 4, pp. 497 - 499
Journal Article
Nature Biotechnology, ISSN 1087-0156, 02/2017, Volume 35, Issue 2, pp. 99 - 100
Journal Article
Trends in Neurosciences, ISSN 0166-2236, 2009, Volume 32, Issue 11, pp. 591 - 601
Histone deacetylases (HDACs) play a key role in homeostasis of protein acetylation in histones and other proteins and in regulating fundamental cellular... 
Neurology | SPINAL MUSCULAR-ATROPHY | DISEASE TRANSGENIC MICE | SUBEROYLANILIDE HYDROXAMIC ACID | PROTECTS DOPAMINERGIC-NEURONS | INCREASES SMN EXPRESSION | VALPROIC ACID | AMYOTROPHIC-LATERAL-SCLEROSIS | HISTONE DEACETYLASE INHIBITORS | BDNF GENE-TRANSCRIPTION | NEUROSCIENCES | HEAT-SHOCK-PROTEIN | Neuroprotective Agents - therapeutic use | Amyotrophic Lateral Sclerosis - physiopathology | Humans | Muscular Atrophy, Spinal - enzymology | Parkinson Disease - drug therapy | Neurodegenerative Diseases - drug therapy | Stroke - physiopathology | Amyotrophic Lateral Sclerosis - drug therapy | Amyotrophic Lateral Sclerosis - enzymology | Acetylation | Huntington Disease - drug therapy | Huntington Disease - enzymology | Muscular Atrophy, Spinal - physiopathology | Huntington Disease - physiopathology | Disease Models, Animal | Alzheimer Disease - physiopathology | Neurodegenerative Diseases - pathology | Alzheimer Disease - drug therapy | Rats | Histone Deacetylases - metabolism | Stroke - drug therapy | Alzheimer Disease - enzymology | Parkinson Disease - physiopathology | Stroke - enzymology | Animals | Neurodegenerative Diseases - physiopathology | Neuroprotective Agents - classification | Parkinson Disease - enzymology | Muscular Atrophy, Spinal - drug therapy | Histone Deacetylase Inhibitors - therapeutic use | Histones - metabolism | Neurodegenerative Diseases - enzymology | Histones | Nervous system diseases | Analysis
Journal Article
Neurology, ISSN 0028-3878, 03/2016, Volume 86, Issue 10, pp. 884 - 885
Spinal muscular atrophy (SMA) is an anterior horn cell disease characterized by severe muscle atrophy and weakness,(1) traditionally divided into 4... 
TRIAL | PHENYLBUTYRATE | FUNCTIONAL MOTOR SCALE | SMN1 | CLINICAL NEUROLOGY | Muscular Atrophy, Spinal - diagnosis | Female | Male | Muscular Atrophy, Spinal - drug therapy | Oligonucleotides - administration & dosage | Humans
Journal Article
Nature Chemical Biology, ISSN 1552-4450, 07/2015, Volume 11, Issue 7, pp. 511 - 517
Journal Article
Lancet Neurology, The, ISSN 1474-4422, 2010, Volume 9, Issue 9, pp. 875 - 884
Journal Article
Developmental Medicine & Child Neurology, ISSN 0012-1622, 01/2019, Volume 61, Issue 1, pp. 19 - 24
Journal Article