making muscle wasting history

What is Duchenne Muscular Dystrophy?

• Last Edited: 22 Jun 2009
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Duchenne muscular dystrophy (Duchenne) is a severe recessive X-linked form of muscular dystrophy characterized by rapid progression of muscle degeneration, eventually leading to paralysis and earl...

Tags: duchenne, duchenne muscular dystophy, inherited, x linked, treatments.

Dystrophin

• Last Edited: 12 Aug 2009
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What is Dystrophin?Dystrophin is a protein which binds to the muscle membrane cells and helps to maintain the muscle cell structure. It acts like a shock absorber for the muscle cells, and enable...

Tags: dystrophin, gene, protein, muscle, muscle cells, duchenne, muscular, dystrophy.

Exon Skipping

• Last Edited: 6 Apr 2009
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The exon skipping technique tries to change a Duchenne mutation into a Becker mutation. If a variation disturbs the reading frame and thus causes Duchenne dystrophy, the reading frame can be resto...

Tags: exon skipping, exon, antisense oligonucleotide, RNA, duchenne.

Utrophin

• Last Edited: 27 May 2009
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Found this interesting article but unfortunately at least 3 years to clinical rials.......http://www.physorg.com/news162569608.htmlNew therapy substitutes missing protein in those with muscular dy...

Tags: utrophin, duchenne, dystrophin, protein, muscle.

Dosing regiment

• Last Edited: 28 May 2009
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this is potentially important ...Dosing regimen has a significant impact on the efficiency of morpholino oligomer-induced exon skipping in mdx mice.http://www.ncbi.nlm.nih.gov/pubmed/194697091: Hu...

Tags: duchenne, dystrophin, dose, peptide, exon skipping, pmo, morpholino.

dmd explanation and information

• Last Edited: 15 Jun 2009
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after spending 4 years trying to find info on how dmd works and what the treatments are, this is the best site for a simple and full explanation.the first link takes to the ygyh.org front page to ...

Tags: duchenne, dmd, genes, genetics.

Holiday Insurance

• Last Edited: 7 Jul 2009
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It is really important that you take out insurance that covers your child’s condition. If you do not declare that they have duchenne it may be invalidated and you will not be able to claim. ...

Tags: insurance, holiday, duchenne, cover.

Duchenne Information

• Last Edited: 26 Jul 2009
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The muscular dystrophies are an inherited group of progressive myopathic disorders resulting from defects in a number of genes required for normal muscle function 1 . The Duchenne and Becker muscu...

Tags: Duchenne, steroids, inhibitors, ace, treatments, heart, lungs.

TREAT-NMD guide to 'Stem Cell Tourism'

• Last Edited: 29 Jan 2010
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TREAT-NMD is aware that new scientific discoveries and innovations can lead to raised expectations and enthusiasm and this may in turn lead to hype, which can be seen as increased publicity and ...

Tags: stem cell tourism, stem cell, tourism, therapies, TREATNMD, hope, hype, neuromuscular disorders, Duchenne, muscular dystrophy, DMD, research.

Blocking the myostatin signal with a dominant negative receptor improves the success of human myoblast transplantation in dystrophin mice.

• Last Edited: 4 Feb 2011
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Duchene muscular dystrophy (DMD) is a recessive disease caused by a dystrophin gene mutation. Myoblast transplantation permits to introduce the dystrophin gene in dystrophic muscle fibers. However...

Tags: gene mutation, myoblasts, Duchenne.

GSK2402968 Clinical Development Programme Update

• Last Edited: 15 Apr 2011
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Forty-Eight Week Follow-Up Data from a Phase I/IIa Extension Study of PRO051/GSK2402968 in Subjects with Duchenne Muscular DystrophyThe following Phase I/IIa extension study results were presented...

Tags: Duchenne, DMD, GSK, Duchenne Muscular Dystrophy.

£2.5m research grant for Duchenne Muscular Dystrophy awarded to Oxford researchers

• Last Edited: 15 Apr 2011
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Funding for pioneering research into gene therapy by Action Duchenne has led to further funds for research into this life limiting disease A recent £2.5 million award made by the Health Innovation...

Tags: exon skipping, Duchenne, Duchenne muscular dystrophy, Dr Matthew Wood, Mike Gait, antisense oligonucleotides.

Update on the Therapeutic approaches to muscular dystrophy

• Last Edited: 10 May 2011
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This is a summary of the article written by Aurelie Goyenvalle, Jane T Seto, Kay E Davies and Jeffrey Chamberlain, which was published by Oxford University Press on 5 April 2011.The article is a r...

Tags: exon skipping, gene therapy, Viral Vector, Dystrophin, rAAV, Antisense Oligonucleotide, PMO, PPMO, Duchenne, muscular dystrophy, Utrophin.