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Treatment of Duchenne Muscular Dystrophy may be Possible Via Molecular Therapy

by Kathy Jones on  July 28, 2011 at 7:35 PM Research News   - G J E 4
People with devastating genetic disease Duchenne muscular dystrophy were found to be benefited by a molecular technique originally developed at the University of North Carolina at Chapel Hill.

Duchenne muscular dystrophy is a lethal disease that affects 1 in 3500 newborn boys.
 Treatment of Duchenne Muscular Dystrophy may be Possible Via Molecular Therapy
Treatment of Duchenne Muscular Dystrophy may be Possible Via Molecular Therapy
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In the disease, omissions or misprints in the letters of the dystrophin gene cause its "reading frame" to shift, abbreviating the instructions for making the dystrophin protein.

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As a result, the cells fail to make a functional muscle protein and patients eventually lose their ability to walk and breathe.

In a study, researchers from the U.K., U.S. and Australia demonstrated that a phase Ib/IIa trial of the approach restored production of the critical muscle protein missing in patients with the progressive neuromuscular condition.

"When I first tried my approach in a test tube some twenty years ago, a reviewer of my manuscript commented that it was 'molecular gymnastics that will never amount to anything,'" said study co-author Ryszard Kole, PhD, a professor of pharmacology who develop the technology with AVI Biopharma, in Bellevue, Washington.

"Now we have evidence that it works, and in an illness that has no other good therapeutic options," he stated.

The novel treatment uses strips of genetic code - called antisense oligonucleotides - to restore the function of a defective dystrophin gene.

The scientists administered the treatment intraveneously (IV) to 19 Duchenne muscular dystrophy patients over the course of 12 weeks.

They found that the drug was well tolerated and appeared to increase the levels of dystrophin protein in a statistically significant dose-dependent manner.

The best 3 responders showed an increase following treatment of protein levels from 2 percent to 18 percent, from 0.9 percent to17 percent and from 0 percent to 7.7 percent of normal muscle, respectively.

The study was published July 25, 2011 in the journal Lancet.

Source: ANI
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my son is involved DMD and his age is 11years ,now his movement is limited .he feel very very diffcult to walk even fell down.i m worry his future. please give me guidance of treatment.
saadi Monday, December 5, 2011
There is no treatment available at present throughout the world. Just give physiotherapy at your home by a trained therapist who knows about DMD. Try to maintain his walking. Once walking is stopped, you cannot bring it back. Don't be cheated by any advertisement who claim that they can cure DMD. visit www.mdfindia.org
mdfindia Friday, March 23, 2012
hi I'm Dr Menat , I'm from Egypt and my son is suffreing from DMD . has this actually been carried out and found to be successful and is is there any possibility of it becoming a near future treatment for DMD? . and Is this treatment for all types of mutations that occur in DMD or for one type only ? . I'm looking forward to hearing your reply soon . Don't forget me .
Mido-93 Wednesday, September 21, 2011
is there any chances of improvement in conditions of patients suffering from DMD??????????
Sushma_Gupta Thursday, September 8, 2011
Dear Sushma, Chances are very less regarding the improvement. Unless some changes occur internally like gene therapy or stemcell therapy from a reliable source, you cannot expect any improvement. Pray to God that the condition should not become worse than it is today. My prayers too are there for all DMD children.
mdfindia Friday, March 23, 2012
hi i am pradnya gaikwad has this actually been carried out and found to be successful and is is there any possibility of it becoming a near future treatment for DMD?
25774230 Tuesday, September 6, 2011
has this actually been carried out and found to be successful and is is there any possibility of it becoming a near future treatment for DMD?
karapinar Saturday, August 27, 2011

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