Friday 27 April 2012

Summit announces approval for Phase 1 Clinical trial - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Summit announces approval for Phase 1 Clinical trial - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Summit Corporation plc (‘Summit’ or ‘the Company’) SUMMIT RECEIVES REGULATORY APPROVAL TO ENTER SMT C1100 INTO A PHASE I CLINICAL TRIAL Oxford, UK, 24 April 2012, Summit (AIM: SUMM), a UK drug discovery company, today announces that its Phase I Clinical Trial Application (‘CTA’) for SMT C1100 has been approved by the UK regulatory body, the Medicines and Healthcare products Regulatory Agency (‘MHRA’).

Thursday 26 April 2012

Action Duchenne supports ground-breaking project at the Royal Holloway, University of London - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Action Duchenne supports ground-breaking project at the Royal Holloway, University of London - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Action Duchenne is to provide �160,000 of funding to support a pioneering project at Royal Holloway, University of London (RHUL). The project, which is being led by Dr Keith Foster and Prof. George Dickson in the School of Biological Sciences at Royal Holloway, is looking at a system to produce full length dystrophin. Dystrophin is the protein that protects and builds muscle, and it is the lack of dystrophin that causes muscular dystrophy. The system has shown dystrophin expressed at the cell membrane which is an indication that it has a therapeutic effect.

Well done to all Action Duchenne London Marathon Runners - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Well done to all Action Duchenne London Marathon Runners - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Congratulations to the 13 people who took part in the London Marathon on Sunday in support of Action Duchenne. A hotter day than expected made conditions challenging for the 35,000 people on the 26.2 mile course but we are happy to report that all Action Duchenne runners looked like they were coping admirably. Well done everyone - please get in contact and let us know how you got on!

If yo'veu been inspired to take part in the London Marathon next year the ballot for places opens on Monday the 30th April, and will fill up within a day. The organisers will contact you by October to let you know if you have been successful. Email kate@actionduchenne.org to register an interest and we will sent you the ballot information.

Action Duchenne supports ground-breaking project at the Royal Holloway, University of London - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Action Duchenne supports ground-breaking project at the Royal Holloway, University of London - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Action Duchenne is to provide �160,000 of funding to support a pioneering project at Royal Holloway, University of London (RHUL). The project, which is being led by Dr Keith Foster and Prof. George Dickson in the School of Biological Sciences at Royal Holloway, is looking at a system to produce full length dystrophin. Dystrophin is the protein that protects and builds muscle, and it is the lack of dystrophin that causes muscular dystrophy. The system has shown dystrophin expressed at the cell membrane which is an indication that it has a therapeutic effect.

Wednesday 25 April 2012

Fling for Logan

On Saturday 12 May 2012 Ryan Linton and Ettienne Viljoen will be undertaking the Tough Mudder challenge to help us to raise funds for Logan.
Tough Mudder events are hardcore 10-12 mile obstacle course with 25 plus obstacles designed by the Special Forces to test your all around strength, stamina, mental grit, and camaraderie. As the leading company in the booming obstacle course industry, Tough Mudder has already challenged half a million inspiring participants worldwide. But Tough Mudder is more than an event, it’s a way of thinking.
Ryan and Ettienne will be putting themselves through this grueling challenge at Boughton House and Estate, Kettering Northhamptonshire.

AVI BioPharma - News Release

AVI BioPharma - News Release: AVI BioPharma to Present Additional Data From the Phase IIb Study of Eteplirsen for the Treatment of Duchenne Muscular Dystrophy at the 2012 AAN Annual Meeting.Findings will be presented today at the American Academy of Neurology 64th Annual Meeting in New Orleans, Louisiana. Principal investigator, Jerry R. Mendell, M.D. of Nationwide Children's Hospital, will describe the data via a brief oral presentation of the abstract titled "A Phase IIb Placebo-Controlled Study of the Exon-Skipping Drug Eteplirsen in Subjects with Duchenne Muscular Dystrophy" during the AAN Emerging Science Session (abstract #004 at 5:54 pm CDT), followed by a more detailed poster presentation (6:30 to 7:00 pm CDT).

Tuesday 24 April 2012

SMT C1100 For Duchenne Muscular Dystrophy Moves To Human Testing - MarketWatch

SMT C1100 For Duchenne Muscular Dystrophy Moves To Human Testing - MarketWatch: The Muscular Dystrophy Association announced today that SMT C1100, an experimental drug for Duchenne muscular dystrophy (DMD), has received approval from regulatory agencies in the United Kingdom to move from laboratory to human testing, starting with healthy human volunteers.


The drug is designed to increase production of a muscle protein utrophin that can potentially compensate for the one that's missing in DMD.

Friday 20 April 2012

Webinar: Results from DuchenneConnect Study - PPMD Community

Webinar: Results from DuchenneConnect Study - PPMD Community: Results from DuchenneConnect Study
DuchenneConnect�and�Parent Project Muscular Dystrophy�are proud to present the next in our ongoing�Direct Access Webinar Series�on�Wednesday, April 25 at 1pm eastern.

Topic:�Results from DuchenneConnect Study
When:�Wednesday, April 25 at 1pm eastern

Thursday 19 April 2012

University of Nevada School of Medicine research discovery and singular focus of April conference signal new hope for patients with rare diseases

Myomatrix 2012 conference to take place from April 22-24: The nation's leading scientists and clinicians exploring treatment breakthroughs for congenital muscular dystrophy will convene April 22-24 on the campus of the University of Nevada, Reno, where research by Dean Burkin has led to a potential therapy.

Thursday 12 April 2012

MDA Funds Development of Utrophin 'Magnet' for DMD/BMD | Quest Magazine Online

MDA Funds Development of Utrophin 'Magnet' for DMD/BMD | Quest Magazine Online: MDA Funds Development of Utrophin 'Magnet' for DMD/BMD
MDA's $1 million grant will help fund the development of TVN-102, which attracts the muscle protein utrophin to the muscle-fiber membrane.

Article Highlights:
  • MDA funding will allow Tivorsan Pharmaceuticals to complete laboratory testing of TVN-102 and, if all goes well, to request permission from the FDA to begin human testing of the drug in 2012 or 2013.
  • TVN-102 (biglycan) acts like a magnet, attracting utrophin to the muscle-fiber membrane. Animal studies suggest that utrophin at the muscle-fiber membrane can partially compensate for a lack of the protein dystrophin at the membrane, helping to stabilize the membrane.

Tuesday 3 April 2012

AVI Summary Announcement - PPMD Community

AVI Summary Announcement - PPMD Community: Results
Results have demonstrated the following:

The group receiving Eteplirsen 30 mg/kg/week over 24 weeks resulted in a 22.5% increase in dystrophin on muscle biopsy, exceeding the company’s expectations. There was no increase in dystrophin in the placebo group.
The group receiving Eteplirsen 50mg/kg/week did not show an increase in dystrophin at 12 weeks, despite the higher dose.
The dystrophin produced appears to be new, novel dystrophin producing muscle fibers of normal length and consistency. The dystrophin appears to be diffusely distributed throughout the muscle biopsy.
There was no change in the clinical outcomes (6 minute walk test, Gower’s maneuver, 10 meter run) in the treatment group at 12 or 24 weeks.
Doses were well tolerated and there were no serious side effects to the drug at either doses.

Monday 2 April 2012

Take Action: Ask Your Representative to Co-Sponsor FAST Act - PPMD Community

Take Action: Ask Your Representative to Co-Sponsor FAST Act - PPMD Community: We are coming to you today to ask that you devote your attention and energy towards legislation that seeks to expedite the process FDA uses to review drug applications. PPMD has officially endorsed�HR. 4132, the Faster Access to Specialized Treatments (FAST) Act, bipartisan legislation that was recently introduced in the House of Representatives.

How you can take action
The FAST Act is consistent with the FDA proposal the PPMD board adopted earlier this year. As such, we are requesting that you – our advocates – contact your members of Congress and urge them to co-sponsor the FAST Act.

We have prepared an email template for you to send to Representatives�and a phone script for additional follow-up.