Featured Research

from universities, journals, and other organizations

Closer to finding treament for Duchenne muscular dystrophy

Date:
December 1, 2011
Source:
University of Royal Holloway London
Summary:
Academics have made an important breakthrough in the development of a treatment for Duchenne muscular dystrophy (DMD).

Dystrophin protein in a human muscle cell.
Credit: Image courtesy of University of Royal Holloway London

Academics from Royal Holloway, University of London and a team lead by scientists at the UCL Institute of Child Health (ICH), have made an important breakthrough in the development of a treatment for Duchenne muscular dystrophy (DMD) which in the future could help the likes of the Lloyd family who were featured on a recent X-Factor T.V. show in the U.K.

The family's story was shown before the X Factor stars performed the show's charity song in aid of sick children. In a moving interview the boys' parents Helen and Chris Lloyd spoke about when their identical twins Dan and Sam, 16, were first diagnosed with the genetic disease DMD when they started showing signs of struggling to move.

But the family's ordeal got worse when younger brother Tom, 13, was also diagnosed with the life-limiting condition that affects one in 3,000 male births in the general population, with around 100 cases diagnosed in the UK each year.

Together with the MDEX Consortium, Professor George Dickson from the School of Biological Sciences at Royal Holloway showed that a gene-based drug treatment was effective in restoring the dystrophin protein that is missing in sufferers of DMD, in seven out of 19 trial participants.

DMD causes progressive muscle weakness due to the breakdown and loss of muscle cells. Patients lack a single important protein in their muscle fibres called dystrophin. By the ages of eight to 12, boys become unable to walk and by their late teens or early twenties the condition can become severe enough to limit life expectancy.

In this clinical trial of 19 patients, study participants aged five to 15 at Great Ormond Street Hospital and the Royal Victoria Infirmary, Newcastle, were given weekly doses of the drug, AVI-4658. The drug had already been tested for safety and efficacy by the MDEX Consortium and AVI Biopharma in an earlier phase of the study (Kinali et al, Lancet Neurol 2009).

Professor Dickson said: "Duchenne dystrophy is a very serious inherited disorder which affects 1 in 3,000 boys from age four onwards so to have all three brothers affected by the disease is very rare indeed. It is a progressive and severe muscle wasting disease which is currently untreatable. But our ongoing clinical trials have great promise as a long-term treatment and we hope in future to be able to help transform the lives of families like the Lloyds who have to go through this terrible ordeal."


Story Source:

The above story is based on materials provided by University of Royal Holloway London. Note: Materials may be edited for content and length.


Cite This Page:

University of Royal Holloway London. "Closer to finding treament for Duchenne muscular dystrophy." ScienceDaily. ScienceDaily, 1 December 2011. <www.sciencedaily.com/releases/2011/11/111128121356.htm>.
University of Royal Holloway London. (2011, December 1). Closer to finding treament for Duchenne muscular dystrophy. ScienceDaily. Retrieved July 30, 2014 from www.sciencedaily.com/releases/2011/11/111128121356.htm
University of Royal Holloway London. "Closer to finding treament for Duchenne muscular dystrophy." ScienceDaily. www.sciencedaily.com/releases/2011/11/111128121356.htm (accessed July 30, 2014).

Share This




More Health & Medicine News

Wednesday, July 30, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Health Insurers' Profits Slide

Health Insurers' Profits Slide

Reuters - Business Video Online (July 30, 2014) Obamacare-related costs were said to be behind the profit plunge at Wellpoint and Humana, but Wellpoint sees the new exchanges boosting its earnings for the full year. Fred Katayama reports. Video provided by Reuters
Powered by NewsLook.com
Concern Grows Over Worsening Ebola Crisis

Concern Grows Over Worsening Ebola Crisis

AFP (July 30, 2014) Pan-African airline ASKY has suspended all flights to and from the capitals of Liberia and Sierra Leone amid the worsening Ebola health crisis, which has so far caused 672 deaths in Guinea, Liberia and Sierra Leone. Duration: 00:43 Video provided by AFP
Powered by NewsLook.com
At Least 20 Chikungunya Cases in New Jersey

At Least 20 Chikungunya Cases in New Jersey

AP (July 30, 2014) At least 20 New Jersey residents have tested positive for chikungunya, a mosquito-borne virus that has spread through the Caribbean. (July 30) Video provided by AP
Powered by NewsLook.com
Xtreme Eating: Your Daily Caloric Intake All On One Plate

Xtreme Eating: Your Daily Caloric Intake All On One Plate

Newsy (July 30, 2014) The Center for Science in the Public Interest released its 2014 list of single meals with whopping calorie counts. Video provided by Newsy
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:

Breaking News:
from the past week

In Other News

... from NewsDaily.com

Science News

Health News

    Environment News

    Technology News



      Save/Print:
      Share:

      Free Subscriptions


      Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

      Get Social & Mobile


      Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

      Have Feedback?


      Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
      Mobile: iPhone Android Web
      Follow: Facebook Twitter Google+
      Subscribe: RSS Feeds Email Newsletters
      Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins