login
Become a member
Quick Registration
Full Registration Benefits -
Free Homepages for Doctors
Free Homepages for Hospitals
Free Homepages for Medical Conference
Free Homepages for NGO's
Personal Health Record Creation
Access to all Medical Slides
Access to all Jobs & Products Sale
New Experimental Drug for Muscular Dystrophy
The condition, Duchenne muscular dystrophy (DMD), occurs among roughly 1 one in 3,500 males.
It occurs because of a deletion in a gene that makes a protein called dystrophin, which helps provide a protective membrane around muscle fibres. Without this skin, muscle fibres become damaged and eventually die.
By they time they are aged eight to 12, boys with DMD become unable to walk and usually face a shortened life expectancy.
In DMD, the problem lies specifically in sections of DNA called exons that, like stepping stones, provide the pathway by which the gene makes dystrophin.
The new drug takes the approach of "exon skipping," or applying a tiny molecular patch over the deletion, so that the gene can produce a shorter but still functional version of the protein.
After successful tests on muscle cells cultured in a lab dish and on mice, the therapy was cautiously tested for safety on 19 ambulatory patients aged five to 15 at hospitals in London and Newcastle.
Seven out the 19 showed a "significant" response, with levels of dystrophin rising to as much as 18 percent of normal levels.
The volunteers, who were treated over 12 weeks, suffered no side effects.
They did not show any major improvement in a standard six-minute walking test, although this was not the goal of the mini-trial.
|
|
|
More News on: Drug Toxicity, Muscular Dystrophy, Signature Drug Toxicity, Genetic Testing of Diseases, Drugs Banned in India
 Comment & Contribute
|
|
sushma2(Guest)
09/01/2011
would there be any result regarding the improvement of those patients who're sufferring from DMD????
Reply |
Forward
|
Medwonders Health Network
- Health News Index
- News Quick Links
 |
Muscular Dystrophy
Complete Medindia Resources
|
| News Categories: |
| » | Schizophrenia Drug Differentiates Cancer Stem Cells into Less Threatening Cells |
| » | Docs Who Seldom Meet Drug Reps Continue to Prescribe Drugs With FDA “Black Box” Warnings |
| » | Revision in Quality Standards for Heparin |
| » | Acne Drug Users At High Risk of Developing Eye Infections |
| » | Patients Who Take Antidepressants When in ICU More Likely to Die |
| More Drug News | |
|
