(Q24234414)

3

Leonard H van den Berg

0 references

Alexander F J E Vrancken

6

Alexander FJE Vrancken

0 references

Susan T. Iannaccone

5

Susan T Iannaccone

0 references

John H. J. Wokke

4

John HJ Wokke

0 references

author name string

Renske I Wadman

1

0 references

Wendy MJ Bosboom

Cochrane Database of Systematic Reviews

2

0 references

publication date

7 December 2011

0 references

published in

0 references

cites work

Randomized, double-blind, placebo-controlled trial of hydroxyurea in spinal muscular atrophy

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Randomized, double-blind, placebo-controlled trial of phenylbutyrate in spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

SMA CARNI-VAL trial part I: double-blind, randomized, placebo-controlled trial of L-carnitine and valproic acid in spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Riluzole pharmacokinetics in young patients with spinal muscular atrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Phenylbutyrate increases SMN gene expression in spinal muscular atrophy patients

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Oral administration of the thyrotropin-releasing hormone (TRH) analogue, taltireline hydrate, in spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Pilot trial of albuterol in spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

The effect of hydroxyurea in spinal muscular atrophy cells and patients

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Pilot trial of phenylbutyrate in spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Role of gabapentin in spinal muscular atrophy: results of a multicenter, randomized Italian study

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Daily salbutamol in young patients with SMA type II

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Phase II open label study of valproic acid in spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Valproic acid treatment in six patients with spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Phenylbutyrate increases SMN expression in vitro: relevance for treatment of spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Salbutamol increases SMN mRNA and protein levels in spinal muscular atrophy cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Neurotrophic factor therapy--prospects and problems

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Autonomic dysfunction in ALS: a preliminary study on the effects of intrathecal BDNF.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Mitochondrial oxidative metabolism in motor neuron degeneration (mnd) mouse central nervous system

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

134th ENMC International Workshop: Outcome Measures and Treatment of Spinal Muscular Atrophy, 11-13 February 2005, Naarden, The Netherlands.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Transport of energy in muscle: the phosphorylcreatine shuttle

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Acetyl-L-carnitine shows neuroprotective and neurotrophic activity in primary culture of rat embryo motoneurons

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Additivity and potentiation of IGF-I and GDNF in the complete rescue of postnatal motor neurons

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Ciliary neurotrophic factor (CNTF) for amyotrophic lateral sclerosis or motor neuron disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Specific antinociceptive activity of cholest-4-en-3-one, oxime (TRO19622) in experimental models of painful diabetic and chemotherapy-induced neuropathy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Critical review of gangliosides and thyrotropin-releasing hormone in peripheral neuromuscular diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Constitutive muscular abnormalities in culture in spinal muscular atrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Thyrotropin-releasing hormone in ALS. Are the results of clinical studies inconsistent?

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Riluzole. A review of its pharmacodynamic and pharmacokinetic properties and therapeutic potential in amyotrophic lateral sclerosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Genetic mapping of chronic childhood-onset spinal muscular atrophy to chromosome 5q11.2-13.3

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Effects of 2,4-diaminoquinazoline derivatives on SMN expression and phenotype in a mouse model for spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Neuroprotective effect of adult hematopoietic stem cells in a mouse model of motoneuron degeneration

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

A double-blind, placebo-controlled trial of TRH in amyotrophic lateral sclerosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Determinants of exon 7 splicing in the spinal muscular atrophy genes, SMN1 and SMN2.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Survival in SMA type I: a prospective analysis of 34 consecutive cases

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

The neurobiology of childhood spinal muscular atrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Clinical trials in spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Histone Deacetylase Inhibition Activity and Molecular Docking of (E )-Resveratrol: Its Therapeutic Potential in Spinal Muscular Atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

A double-blind, placebo-controlled randomized clinical trial of alpha-tocopherol (vitamin E) in the treatment of amyotrophic lateral sclerosis. ALS riluzole-tocopherol Study Group

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Genetic conversion of an SMN2 gene to SMN1: a novel approach to the treatment of spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Chaos in the classification of SMA: a possible resolution.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

The role of creatine in the management of amyotrophic lateral sclerosis and other neurodegenerative disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Quantitative analyses of SMN1 and SMN2 based on real-time lightCycler PCR: fast and highly reliable carrier testing and prediction of severity of spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Neuronal SMN expression corrects spinal muscular atrophy in severe SMA mice while muscle-specific SMN expression has no phenotypic effect

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Spinal muscular atrophy: molecular pathophysiology

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Diet- and valproate-induced transient hyperammonemia: effect of L-carnitine.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

High dose vitamin E therapy in amyotrophic lateral sclerosis as add-on therapy to riluzole: results of a placebo-controlled double-blind study

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Possible strategies for treatment of SMA patients: a neurobiologist's view

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

A randomized sequential trial of creatine in amyotrophic lateral sclerosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Hydroxyurea enhances SMN2 gene expression in spinal muscular atrophy cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Use of TRH analogues in motorneurone disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Gene therapy of murine motor neuron disease using adenoviral vectors for neurotrophic factors

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Riluzole attenuates spinal muscular atrophy disease progression in a mouse model.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Tetracyclines that promote SMN2 exon 7 splicing as therapeutics for spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Reliability of 4 outcome measures in pediatric spinal muscular atrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

The role of SMN in spinal muscular atrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Reduced SMN protein impairs maturation of the neuromuscular junctions in mouse models of spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

The role of histone acetylation in SMN gene expression.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Anti-glutamatergic effect of riluzole: comparison with valproic acid

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Do inhaled beta(2)-agonists have an ergogenic potential in non-asthmatic competitive athletes?

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

L-carnitine suppresses the onset of neuromuscular degeneration and increases the life span of mice with familial amyotrophic lateral sclerosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Randomized, double-blind, placebo-controlled trial of albuterol in facioscapulohumeral dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Creatine for treating muscle disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Muscle strength and motor function in children and adolescents with spinal muscular atrophy II and III.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Identification and characterization of a spinal muscular atrophy-determining gene

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Correlation between severity and SMN protein level in spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

21 January 2018

Spinal muscular atrophy.

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Knockdown of the survival motor neuron (Smn) protein in zebrafish causes defects in motor axon outgrowth and pathfinding.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Efficacy and safety of xaliproden in amyotrophic lateral sclerosis: results of two phase III trials

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Connecting impairment, disability, and handicap in immune mediated polyneuropathies

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

90th ENMC international workshop: European Spinal Muscular Atrophy Randomised Trial (EuroSMART) 9-10 February 2001, Naarden, The Netherlands

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Pilot trial of salbutamol in central core and multi-minicore diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Clinical trials of riluzole in patients with ALS. ALS/Riluzole Study Group-II.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Placebo-controlled trial of gabapentin in patients with amyotrophic lateral sclerosis. WALS Study Group. Western Amyotrophic Lateral Sclerosis Study Group

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

A placebo-controlled trial of recombinant human ciliary neurotrophic (rhCNTF) factor in amyotrophic lateral sclerosis. rhCNTF ALS Study Group

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

A placebo-controlled trial of gabapentin in spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Phase III randomized trial of gabapentin in patients with amyotrophic lateral sclerosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND)

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Intrathecal thyrotropin-releasing hormone does not alter the progressive course of ALS: experience with an intrathecal drug delivery system

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Selective vulnerability of motor neurons and dissociation of pre- and post-synaptic pathology at the neuromuscular junction in mouse models of spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Spinal muscular atrophy: recent advances and future prospects.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Plastin 3 is a protective modifier of autosomal recessive spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Survival motor neuron protein regulates apoptosis in an in vitro model of spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Intragenic telSMN mutations: frequency, distribution, evidence of a founder effect, and modification of the spinal muscular atrophy phenotype by cenSMN copy number.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

A novel function for SMN, the spinal muscular atrophy disease gene product, in pre-mRNA splicing

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

An analysis of extended survival in patients with amyotrophic lateral sclerosis treated with riluzole

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Drug treatment for facioscapulohumeral muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Delivery of recombinant follistatin lessens disease severity in a mouse model of spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Sodium valproate exerts neuroprotective effects in vivo through CREB-binding protein-dependent mechanisms but does not improve survival in an amyotrophic lateral sclerosis mouse model

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Spinal muscular atrophy: new thoughts on the pathogenesis and classification schema

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

A Phase 1 Trial of Riluzole in Spinal Muscular Atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

A double-blind randomized clinical trial in amyotrophic lateral sclerosis using lamotrigine: effects on CSF glutamate, aspartate, branched-chain amino acid levels and clinical parameters

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Creatine monohydrate in DM2/PROMM: a double-blind placebo-controlled clinical study. Proximal myotonic myopathy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

A clinical trial of creatine in ALS.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Valproic acid increases SMN levels in spinal muscular atrophy patient cells.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Molecular mechanisms of spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Natural history of denervation in SMA: relation to age, SMN2 copy number, and function

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Efficacy of thyrotropin-releasing hormone in the treatment of spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Creatine monohydrate increases strength in patients with neuromuscular disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

A summary of mechanistic hypotheses of gabapentin pharmacology

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

The natural history of type I (severe) spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Synthesis and biological evaluation of novel 2,4-diaminoquinazoline derivatives as SMN2 promoter activators for the potential treatment of spinal muscular atrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

An outcome study of riluzole in amyotrophic lateral sclerosis--a population-based study in Ireland, 1996-2000.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Effects of training and albuterol on pain and fatigue in facioscapulohumeral muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Consensus Statement for Standard of Care in Spinal Muscular Atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Spinal muscular atrophy: from gene to therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Mildly affected patients with spinal muscular atrophy are partially protected by an increased SMN2 copy number

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Treatment for Charcot-Marie-Tooth disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

5-(N-ethyl-N-isopropyl)-amiloride enhances SMN2 exon 7 inclusion and protein expression in spinal muscular atrophy cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Natural history in proximal spinal muscular atrophy. Clinical analysis of 445 patients and suggestions for a modification of existing classifications

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

59th ENMC International Workshop: Spinal Muscular Atrophies: recent progress and revised diagnostic criteria 17-19 April 1998, Soestduinen, The Netherlands.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

SMN protects cells against mutant SOD1 toxicity by increasing chaperone activity

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Combined effects of GDNF, BDNF, and CNTF on motoneuron differentiation in vitro

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

A study of thyrotropin-releasing hormone for the treatment of spinal muscular atrophy: a preliminary report

21 January 2018

2 references

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

21 January 2018

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

A Randomized, Placebo-Controlled Trial of Creatine in Children With Spinal Muscular Atrophy

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

A pilot trial with clenbuterol in amyotrophic lateral sclerosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Genetic homogeneity between acute and chronic forms of spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

Gene for chronic proximal spinal muscular atrophies maps to chromosome 5q

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

In vivo activation of SMN in spinal muscular atrophy carriers and patients treated with valproate

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

Spinal muscular atrophy and therapeutic prospects

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

Carnitine and acyltransferase in experimental neurogenic atrophies: changes with treatment

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

Correlation between SMN2 copy number and clinical phenotype of spinal muscular atrophy: three SMN2 copies fail to rescue some patients from the disease severity

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

A natural history study of late onset spinal muscular atrophy types 3b and 4.

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

Mapping of acute (type I) spinal muscular atrophy to chromosome 5q12-q14

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

Prospective study of spinal muscular atrophy before age 6 years. DCN/SMA Group

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

Two successful double-blind trials with coenzyme Q10 (vitamin Q10) on muscular dystrophies and neurogenic atrophies

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

A collaborative study on the natural history of childhood and juvenile onset proximal spinal muscular atrophy (type II and III SMA): 569 patients

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

The natural history and the effects of gabapentin in amyotrophic lateral sclerosis

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

Dose-ranging study of riluzole in amyotrophic lateral sclerosis. Amyotrophic Lateral Sclerosis/Riluzole Study Group II

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

International SMA consortium meeting. (26-28 June 1992, Bonn, Germany)

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

Spinal muscular atrophy

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

SMN oligomerization defect correlates with spinal muscular atrophy severity

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

Salbutamol, a beta 2-adrenoceptor agonist, increases skeletal muscle strength in young men

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

Spinal muscular atrophy

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

A controlled trial of riluzole in amyotrophic lateral sclerosis. ALS/Riluzole Study Group

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

Skipping toward personalized molecular medicine

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

Local dystrophin restoration with antisense oligonucleotide PRO051

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

Protective effects of cardiotrophin-1 adenoviral gene transfer on neuromuscular degeneration in transgenic ALS mice

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

Neurofilament accumulation at the motor endplate and lack of axonal sprouting in a spinal muscular atrophy mouse model

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

The role of the SMN gene in proximal spinal muscular atrophy

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

Therapeutic benefits of cardiotrophin-1 gene transfer in a mouse model of spinal muscular atrophy

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

De novo and inherited deletions of the 5q13 region in spinal muscular atrophies

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

Valproate may improve strength and function in patients with type III/IV spinal muscle atrophy

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

Controlled trial of thyrotropin releasing hormone in amyotrophic lateral sclerosis

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

Amyotrophic lateral sclerosis: effects of acute intravenous and chronic subcutaneous administration of thyrotropin-releasing hormone in controlled trials

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

A double-blind placebo-controlled clinical trial of subcutaneous recombinant human ciliary neurotrophic factor (rHCNTF) in amyotrophic lateral sclerosis. ALS CNTF Treatment Study Group

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

Function changes in spinal muscular atrophy II and III. The DCN/SMA Group

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

A controlled trial of recombinant methionyl human BDNF in ALS: The BDNF Study Group (Phase III)

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

Creatine monohydrate in muscular dystrophies: A double-blind, placebo-controlled clinical study

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021

Homozygous deletion of the survival motor neuron 2 gene is a prognostic factor in sporadic ALS

1 reference

https://api.crossref.org/works/10.1002%2F14651858.CD006282.PUB3

7 January 2021