Candidate Proteins, Metabolites and Transcripts in the Biomarkers for Spinal Muscular Atrophy (BforSMA) Clinical Study

Evaluation of SMN Protein, Transcript, and Copy Number in the Biomarkers for Spinal Muscular Atrophy (BforSMA) Clinical Study

(2012) Thomas O. Crawford et al.   PLoS One

Moving towards meaningful measurement: Rasch analysis of the North Star Ambulatory Assessment in Duchenne muscular dystrophy

(2011) ANNA MAYHEW et al.   DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY

Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy

(2011) M. A. Passini et al.   Science Translational Medicine

EGFR gene copy number as a predictive biomarker for patients receiving tyrosine kinase inhibitor treatment: a systematic review and meta-analysis in non-small-cell lung cancer

(2010) I. J. Dahabreh et al.   ANNALS OF ONCOLOGY

Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice

(2010) Elisa Dominguez et al.   HUMAN MOLECULAR GENETICS

CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy

(2010) Marco A. Passini et al.   JOURNAL OF CLINICAL INVESTIGATION

Semi-Targeted Plasma Proteomics Discovery Workflow Utilizing Two-Stage Protein Depletion and Off-Line LC−MALDI MS/MS

(2010) Peter Juhasz et al.   JOURNAL OF PROTEOME RESEARCH

Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

(2010) Kevin D Foust et al.   NATURE BIOTECHNOLOGY

Systemic Delivery of scAAV9 Expressing SMN Prolongs Survival in a Model of Spinal Muscular Atrophy

(2010) C. F. Valori et al.   Science Translational Medicine

Rethinking ALS: The FUS about TDP-43

(2009) Clotilde Lagier-Tourenne et al.   CELL

Biochemical and genetic evidence for a role of IGHMBP2 in the translational machinery

(2009) Mariàngels de Planell-Saguer et al.   HUMAN MOLECULAR GENETICS

RNA processing defects associated with diseases of the motor neuron

(2009) Stephen J. Kolb et al.   MUSCLE & NERVE

Phase II Open Label Study of Valproic Acid in Spinal Muscular Atrophy

(2009) Kathryn J. Swoboda et al.   PLoS One

Reduced expression of the Kinesin-Associated Protein 3 (KIFAP3) gene increases survival in sporadic amyotrophic lateral sclerosis

(2009) J. E. Landers et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Tetracyclines That Promote SMN2 Exon 7 Splicing as Therapeutics for Spinal Muscular Atrophy

(2009) M. L. Hastings et al.   Science Translational Medicine

The Liver Toxicity Biomarker Study: Phase I Design and Preliminary Results

(2009) Robert N. Mcburney et al.   TOXICOLOGIC PATHOLOGY

Oxidative stress biomarkers in sporadic ALS

(2008) Hiroshi Mitsumoto et al.   Amyotrophic Lateral Sclerosis

The effect of hydroxyurea in spinal muscular atrophy cells and patients

(2008) Wen-Chen Liang et al.   JOURNAL OF THE NEUROLOGICAL SCIENCES

Enhanced Detection of Low Abundance Human Plasma Proteins Using a Tandem IgY12-SuperMix Immunoaffinity Separation Strategy

(2008) Wei-Jun Qian et al.   MOLECULAR & CELLULAR PROTEOMICS

Correlation network analysis for data integration and biomarker selection

(2008) Aram Adourian et al.   Molecular BioSystems

Gene expression in blood of subjects with Duchenne muscular dystrophy

(2008) Brenda Wong et al.   NEUROGENETICS

A CSF biomarker panel for identification of patients with amyotrophic lateral sclerosis

(2008) R. M. Mitchell et al.   NEUROLOGY

Spinal muscular atrophy

(2008) Maryam Oskoui et al.   Neurotherapeutics