SMA valiant trial: A prospective, double-blind, placebo-controlled trial of valproic acid in ambulatory adults with spinal muscular atrophy

Mouse models of SMA: tools for disease characterization and therapeutic development

(2012) Thomas W. Bebee et al.   HUMAN GENETICS

Childhood spinal muscular atrophy: controversies and challenges

(2012) Eugenio Mercuri et al.   LANCET NEUROLOGY

Sense in Antisense Therapy for Spinal Muscular Atrophy

(2012) Alex MacKenzie   NEW ENGLAND JOURNAL OF MEDICINE

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

(2012) Richard S. Finkel et al.   PLoS One

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

Spinal Muscular Atrophy

(2011) Stephen J. Kolb   ARCHIVES OF NEUROLOGY

Evaluation of muscle strength and motor abilities in children with type II and III spinal muscle atrophy treated with valproic acid

(2011) Illora A Darbar et al.   BMC Neurology

Spinal Muscular Atrophy: A Clinical and Research Update

(2011) Jennifer A. Markowitz et al.   PEDIATRIC NEUROLOGY

SMA CARNI-VAL TRIAL PART II: A Prospective, Single-Armed Trial of L-Carnitine and Valproic Acid in Ambulatory Children with Spinal Muscular Atrophy

(2011) John T. Kissel et al.   PLoS One

Antisense oligonucleotides and spinal muscular atrophy: skipping along

(2010) A. H. M. Burghes et al.   GENES & DEVELOPMENT

Spinal muscular atrophy: mechanisms and therapeutic strategies

(2010) C. L. Lorson et al.   HUMAN MOLECULAR GENETICS

Adiposity is increased among high-functioning, non-ambulatory patients with spinal muscular atrophy

(2010) Douglas M. Sproule et al.   NEUROMUSCULAR DISORDERS

SMA CARNI-VAL Trial Part I: Double-Blind, Randomized, Placebo-Controlled Trial of L-Carnitine and Valproic Acid in Spinal Muscular Atrophy

(2010) Kathryn J. Swoboda et al.   PLoS One

A Positive Modifier of Spinal Muscular Atrophy in the SMN2 Gene

(2009) Thomas W. Prior et al.   AMERICAN JOURNAL OF HUMAN GENETICS

An analysis of disease severity based on SMN2 copy number in adults with spinal muscular atrophy

(2009) Bakri Elsheikh et al.   MUSCLE & NERVE

Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick?

(2009) Arthur H. M. Burghes et al.   NATURE REVIEWS NEUROSCIENCE

Increased fat mass and high incidence of overweight despite low body mass index in patients with spinal muscular atrophy

(2009) Douglas M. Sproule et al.   NEUROMUSCULAR DISORDERS

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

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

Multiple therapeutic effects of valproic acid in spinal muscular atrophy model mice

(2008) Li-Kai Tsai et al.   JOURNAL OF MOLECULAR MEDICINE-JMM