Sophie Patmore - Mitochondrial Disease - Long Term Ventilation - Tracheostomy
 
LATEST NEWS
01/08/2010 07:37:19
Thank you to 'Stitches of Love and Kindness'
A massive thank you to everyone at 'Stiches of Love and Kindness' who helped to make Sophie's beautiful handmade quilt.
Read More >>
06/04/2010 19:17:04
Thank you to Jaxson's Blankies for Babes
A special thank you to Jaxson's Blankies for Babes for the special blanket sent to Sophie
Read More >>
14/12/2009 21:45:16
The Holly Bush launch new website
The Holly Bush country pub based in Frensham launch their own website.
Read More >>
View All News >>

RECENT BLOGS
01/09/2010 21:37:57
Sophie loves her new train set!
Sophie has lots of fun playing with her new switch adapted Thomas the Tank Engine train set
Read Blog >>
16/08/2010 21:51:39
Lots of Water Fun and an Energy Crash
Sophie had a great July with lots of tomboy fun. Unfortunately the end of July and early August bought with it an energy crash.
Read Blog >>
View All Blogs >>

LATEST NEWS

<< Back to Previous Page
21/04/2009 15:45:00
spacerImage
Muscular dystrophy gene therapy created
spacerImage

scientists say they've conducted the first gene therapy trial for a form of muscular dystrophy that disables by weakening hip and shoulder muscles.

Researchers from the University of Florida, Nationwide Children's Hospital in Columbus, Ohio, and Ohio State University said they safely transferred a gene to produce a protein necessary for healthy muscle fiber growth into three teenagers with limb-girdle muscular dystrophy.

"We think this is an important milestone in establishing the successful use of gene therapy in muscular dystrophy," said Dr. Jerry Mendell, lead author of the study. "This trial sets the stage for moving forward with treatment for this group of diseases and we are very pleased with these promising initial results.

"In subsequent steps we plan to deliver the gene through the circulation in hopes of reaching multiple muscles. We also want to extend the trials over longer time periods to be sure of the body's reaction," said Mendell.

Limb-girdle muscular dystrophy actually describes more than 19 disorders that occur because patients have a faulty alpha-sarcoglycan gene. In each of the disorders, the muscle fails to produce a protein essential for muscle fibers to thrive. It can occur in children or adults.

The study's findings are to appear in the journal Annals of Neurology.

spacerImage
 
 
HOME PAGE | SOPHIE'S STORY | OUR PHOTOS | RECENT BLOGS | MEDICAL INFO | USEFUL LINKS | CONTACT US | ANTIVIRUS FIREWALL SOFTWARE | FORUM
SOPHIE PATMORE'S STORY - © COPYRIGHT 2010 - ALL RIGHTS RESERVED.

WEB DESIGN SURREY