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PLoS ONE, ISSN 1932-6203, 11/2013, Volume 8, Issue 11, p. e80263
Journal Article
Lancet Neurology, The, ISSN 1474-4422, 2017, Volume 16, Issue 7, pp. 513 - 522
Journal Article
Human Mutation, ISSN 1059-7794, 06/2012, Volume 33, Issue 6, pp. 981 - 988
Journal Article
Molecular Therapy - Nucleic Acids, ISSN 2162-2531, 2016, Volume 5, Issue 7, pp. e331 - e331
The identification of noninvasive biomarkers to monitor the disease progression in spinal muscular atrophy (SMA) is becoming increasingly important. MicroRNAs... 
antisense oligonucleotides therapy | microRNA | spinal muscular atrophy | biomarker | Antisense oligonucleotides therapy | micro RNA | CIRCULATING MICRORNAS | SURVIVAL | MEDICINE, RESEARCH & EXPERIMENTAL | SMA MICE | MOTOR-NEURON | BIOMARKERS | RESCUE | OLIGOMER | MOUSE MODEL | DISEASE | EXPRESSION | Biomarkers | Therapy | Spine | Medical disorders | MicroRNAs | Original
Journal Article
Journal Article
PLoS ONE, ISSN 1932-6203, 02/2017, Volume 12, Issue 2, pp. e0172346 - e0172346
Journal Article
Journal Article
Journal Article
Muscle & Nerve, ISSN 0148-639X, 04/2019, Volume 59, Issue 4, pp. 426 - 430
Introduction: The aim of the study was to assess 12 month changes in upper limb function in patients affected by spinal muscular atrophy type 2 and 3. Methods:... 
Spinal muscular atrophy | Atrophy | Muscles | Patients | Modules
Journal Article
Journal Article
Nature Genetics, ISSN 1061-4036, 12/2011, Volume 43, Issue 12, pp. 1189 - 1193
Journal Article
Journal Article
Journal Article