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Articles tagged with Duchenne muscular dystrophy

Inhibition of TGF-beta

The transforming growth factor beta (TGFβ) is a protein which inhibits the satellite cells (muscle stem cells) from regenerating muscle tissue. Mdx mice and also Duchenne boys have increa...

Tags: Duchenne Muscular Dystrophy, fibrosis, mdx mice, pirfenidone, TGF946.


For a child with Duchenne the primary school years are usually a time of significant change in terms of their independent mobility. When particular changes occur is a little variable but the patte...

Tags: Duchenne muscular dystrophy, MOBILITY, WEAKness in the thighs, physical activies.

2-'O-methyl antisense oligos (PS) targeting exon 44.

BackgroundAntisense-mediated exon skipping is a putative treatment for Duchenne muscular dystrophy (DMD). Using antisense oligonucleotides (AONs), the disrupted DMD reading frame is restored, allo...

Tags: antisense oligonucleotides, Duchenne muscular dystrophy, exon skipping, 28242Omethyl phosphorothioate, phosphorodiamidate morpholino oligomer.

By-passing the nonsense mutation in the 4 CV mouse model of muscular dystrophy by induced exon skipping.

BackgroundDuchenne muscular dystrophy (DMD), a severe neuromuscular disorder, is caused by protein-truncating mutations in the dystrophin gene. Absence of functional dystrophin renders muscle fibr...

Tags: antisense oligomer, B6RosCgDmdmdx4CvJ 4CV mouse, Duchenne muscular dystrophy, exon skipping, morpholino.

An intronic LINE-1 element insertion in the dystrophin gene aborts dystrophin expression and results in duchenne-like muscular dystrophy in the corgi breed

Duchenne muscular dystrophy (DMD) is a dystrophin-deficient lethal muscle disease. To date, the catastrophic muscle wasting phenotype has only been seen in dystrophin-deficient human and dogs. Alt...

Tags: dystrophin, Duchenne muscular dystrophy, DMD.

Targeting Fibrosis in Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is the most common genetic muscle disease affecting 1 in 3,500 live male births. It is an X-linked recessive disease caused by a defective dystrophin gene. The di...

Tags: Duchenne muscular dystrophy, Fibrosis, muscle, cell therapies.

Interleukin-10 reduces the pathology of mdx muscular dystrophy by deactivating M1 macrophages and modulating macrophage phenotype.

Molecular, Cellular & Integrative Physiology Program, University of California, Los Angeles, CA.M1 macrophages play a major role in worsening muscle injury in the mdx mouse model of Duchenne m...

Tags: mdx muscles, dystrophy, Duchenne muscular dystrophy.

Duchenne muscular dystrophy - What causes the increased membrane permeability in skeletal muscle?

Duchenne muscular dystrophy is a severe muscle wasting disease caused by a mutation in the gene for dystrophin – a cytoskeletal protein connecting the contractile machinery to a group of pro...

Tags: muscle weakness, atrophy, therapies, Duchenne muscular dystrophy.

GSK2402968 Clinical Development Programme Update

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

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.

Oligos and Oxygen

In their recent publication Action Duchenne’s Boys to Men campaign documents very well the exciting developments in genetic therapies for Duchenne Muscular Dystrophy.Clinical trials for Exon...

Tags: Duchenne, Duchenne Muscular Dystrophy, Boys to Men, gene therapy, respiratory management, neuromuscular, campaign.

Utrophin expression and muscle regeneration - new studies

From Dr Karl Bettelheim Trustee of Action Duchenne. You can view all Karls reviews at http://www.actionduchenne.org/stopwasting/bettelheim/newsIntroduction: In an earlier review from last year (No...

Tags: dystropin, duchenne, utrophin, gene therapy, duchenne muscular dystrophy, muscular dystrophy.

Skipping with viruses

Research Review No. 56. by Dr Karl Bettelheim Action Duchenne Trustee. You can read all of Karl’s reviews at http://www.actionduchenne.org/stopwasting/bettelheim/newsIntroduction: Exon-skip...

Tags: exon skipping, gene therapy, skipDuchenne, viral vector, therapy, treatment, duchenne, muscular dystrophy, duchenne muscular dystrophy.


Article by Dr Karl Bettelheim Action Duchenne Trustee. You can see all Karl’s reviews at http://www.actionduchenne.org/stopwasting/bettelheim/newsIntroduction: In an earlier review from last...

Tags: melatonin, duchenne, duchenne muscular dystrophy, muscular dystrophy, anti oxident.

Exon skipping for Duchenne Muscular Dystrophy: a successful clinical trial in the UK

By Professor Francesco Muntoni, UCL Institute of Child Health LondonProfessor Francesco Muntoni of UCL and Head of the MDEX Consortium discusses the results of the latest successful experimental t...

Tags: Exon skipping, AVI BioPharma, AVI4658, antisense oligonucleotide, exon 51, duchenne, muscular dystrophy, duchenne muscular dystrophy.

Exon skipping drug benefits Duchenne patients in Clinical Trial

The US biopharma Sarepta (previuosly AVI Biopharma)released important data relating to their extended trial using the morpholino backbone chemistry exon skipping drug Eteplirsen.Serapta News relea...

Tags: duchenne, duchenne muscular dystrophy, exon skipping, clinical trials, sarepta.

An open letter from Chris Garabedian, president and CEO of Sarepta Therapeutics

Action Duchenne reviewed the interim report from Sarepta on the progress of their clinical trial for the exon skipping drug Eteplirsen. see Exon skipping drug benefits Duchenne patients in Clinica...

Tags: duchenne, duchenne muscular dystrophy, Sarepta, eteplirsen, clinical trials, action duchenne.


Introduction: Since the studies by Watson and Crick in the 1950’s, it has been established that Deoxyribonucleic Acid (DNA) is the basis of the characteristics of all life forms on this plane...

Tags: duchenne, duchenne muscular dystrophy, genes, genetics, DNA.

Sarepta announce very promising results from clinical trial for Eteplirsen to treat Duchenne Muscular Dystrophy

Sarepta have announced very promising data relating to their clinical trial for exon skipping drug eteplirsen. The current formulation skips exon 51 to produce a dystrophin protein. See previous r...

Tags: Duchenne, Duchenne Muscular Dystrophy, Sarepta, exon skipping.

Duchenne Muscular Dystrophy and ADHD

Action Duchenne families and throughout our Conferences have reported higher than expected incidences of ADHD in the Duchenne population.A recent research paper from Italy comments “Our res...

Tags: ADHD, Duchenne, Duchenne Muscular Dystrophy, ritalin, patient voices.

Prosensa announces further exon skipping drugs for Duchenne Muscular Dystrophy

Prosensa from the Netherlands have announced their programme for taking Exon Skipping forward. The Dutch company in collaboration with GSK have developed an exon skipping drug based upon 2’O...

Tags: duchenne, exon skipping, duchenne muscular dystrophy, prosensa, gsk, oligonucleotides, clinical trials.