Drug treatment for spinal muscular atrophy types II and III (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

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

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

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

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

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Consensus Statement for Standard of Care in Spinal Muscular Atrophy

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

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

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

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

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SMN protects cells against mutant SOD1 toxicity by increasing chaperone activity

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

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

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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