Monday 27 August 2012

Beyond Exon 51 - PPMD Community

Beyond Exon 51 - PPMD Community: As Sarepta (formely AVI) announces promising results from its phase II interim data�and GSK follows up its promising phase II data with a phase III study, things are looking good for an exon-skipping based therapy for Duchenne…if you happen to be in the 13% of those with Duchenne who have a mutation that can be improved by skipping exon 51. For quick reference, this group includes out-of-frame deletions in exons 45-50, 47-50, 48-50, 49-50, 50, and 52.

Thursday 16 August 2012

Father kayaks across the English Channel to raise funds to fight son’s illness - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Father kayaks across the English Channel to raise funds to fight son’s illness - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Father from Dibden Purlieu, Stuart Littler, is to kayak across the English channel with his two friends, Charlie Brett and Jon Turner to raise funds to find viable treatments for his son, Ben. Ben, who is 6 in September, was diagnosed with Duchenne Muscular Dystrophy in December 2009. Since then the family have been working with charity Action Duchenne to raise money to improve awareness and funds for this life limiting condition. The three friends are novice kayakers and have never attempted anything like this before. In the process they have already met their initial target of raising �5000, and are now aiming for �10,000, to be donated to Action Duchenne. The crossing is set to take place during week commencing 27 August, and will be dependent on tides and weather.

Friday 10 August 2012

Sun News : New research shows hope of cure for muscular dystrophy

Sun News : New research shows hope of cure for muscular dystrophy: EDMONTON -- There is hope in sight for those suffering from muscular dystrophy after a breakthrough discovery at the University of Alberta that could someday cure the disease.

Dr. Toshifumi Yokota, a researcher at the university's faculty of medicine and dentistry, has been working on a gene treatment therapy for over five years that targets the gene responsible for causing Duchenne muscular dystrophy.

Thursday 9 August 2012

Progress on Duchenne muscular dystrophy via antisense exon region skipping

Progress on Duchenne muscular dystrophy via antisense exon region skipping: new work by a Japanese team that shows a promising approach in an animal model of Duchenne muscular dystrophy, the most common form of the disease.

Thursday 2 August 2012

Ryan Lochte - End Duchenne: Ryan Lochte

Ryan Lochte - End Duchenne: Ryan Lochte: Olympic Champion Ryan Lochte lost a member of his family to Duchenne muscular dystrophy, a fatal disease that robs boys of their muscle movement. Ryan is dedicated to the fight to end Duchenne and hopes you will join him.