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King's College London - Health: More than a medical matter

Treatment hope for childhood muscle wasting disorder

24 September 2007

By Ailsa Stevens

Appeared in BioNews 426

The first trial of a new treatment for Duchenne muscular dystrophy (DMD) will start later this year in the UK. The treatment, which has been developed by using human cells and mice experiments, hopes to overcome the effects of the genetic defect that causes the muscle wasting disorder. The plans were announced last week at the annual conference of the British Society for Human Genetics (BSHG) by Professor Francesco Muntoni, a Paediatric Neurologist at Imperial College's School of Medicine, who led the research.

DMD is a muscle-wasting disease that causes a steady deterioration of muscles and often results in death before the age of 30. The condition, which is usually inherited and incurable, affects 1 in 3500 male newborn boys. According to the researchers, DMD is caused by an inherited fault in the gene that encodes the body's instructions for making dystrophin, a crucial muscle protein.

Working in partnership with the Muscular Dystrophy Campaign, the Duchenne Parent Project and Duchenne Support Group, the scientists in this UK consortium (the MDEX Consortium) will use 'antisense-RNA' - a chemical relative of DNA - as a 'molecular patch' to fix the dystrophin gene deletions that are frequently the cause of DMD.

Professor Muntoni said: 'We knew from previous studies in mice that faulty dystrophin genes could be mostly repaired in this way, returning muscle strength to about 70 per cent of normal. However, this is the first time that different research groups have teamed up to pinpoint the optimal target DNA sequences in DMD that could be used in the treatment of humans'.

The initial trial will just explore the safety and efficacy of the new therapy by injecting the antisense RNA molecule into one muscle, but Professor Muntoni is optimistic that the same treatment could also be administered systemically, to target all the muscles of the body.


'Patch' bid to treat muscle disease
Press Association | 18 September 2007
'Patch' test for boys' wasting disease
The Times | 17 September 2007


09 September 2013 - by James Brooks 
A parliamentary report has recommended a ring-fenced fund to ensure access to 'low volume, high cost' treatments for rare diseases... [Read More]
13 June 2011 - by Sarah Guy 
The NHS must be prepared to screen every newborn baby for Duchenne muscular dystrophy (DMD) – the most severe form of muscular dystrophy – in three years time, says a leading UK geneticist who explains that promising treatments are close to becoming a reality... [Read More]
15 November 2009 - by Alison Cranage 
Research published in the journal Science Translational Medicine last week shows gene therapy can improve muscle size and strength in monkeys. The technique holds promise as a therapy for several neuromuscular disorders, and researchers hope that clinical trials will start next year.... [Read More]
27 January 2008 - by Dr Rebecca Robey 
Researchers at the University of Texas Southwestern Medical Centre have developed a new technique to treat the symptoms of muscular dystrophy using embryonic stem (ES) cells. The group, reporting in the February 2008 issue of Nature Medicine, successfully manipulated mouse ES cells to transform them into muscle-forming... [Read More]
07 January 2008 - by Ailsa Stevens 
An experimental treatment for boys with the inherited muscle wasting disease Duchene Muscular Dystrophy (DMD) has showed promise in human safety trials, according to a study published in the New England Journal of Medicine. In the first ever trial on humans, the new drug was shown to... [Read More]

30 April 2007 - by Heidi Nicholl 
A new drug is being trialled in humans which has been able to cure Duchenne muscular dystrophy (DMD) in mice with symptoms of the disease. The drug, named PTC124, has been developed by scientists working at the University of Massachusetts Medical School and Pennsylvania University Medical School... [Read More]
19 November 2006 - by Heidi Nicholl 
A team of scientists based in France and Italy have published work showing a huge improvement in the condition of dogs suffering from a canine version of muscular dystrophy when treated with adult stem cells. Duchenne muscular dystrophy is a serious disease that affects 1 in every... [Read More]
30 March 2006 - by BioNews 
US researchers have begun a clinical trial to test a new gene therapy treatment for Duchenne muscular dystrophy (DMD), an inherited muscle-wasting disorder. The team, based at the Columbus Children's Hospital in Ohio, will test the safety and effectiveness of gene injections in six affected boys aged 8-12 years... [Read More]

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