Wednesday 30 May 2012

Tough Mudder completed!

On 12 May Ryan & Ettienne completed their tough mudder challenge and have surpassed their fundraising target for Logan - many thanks guys.

I will post photos as soon as I get them.

Tuesday 29 May 2012

Opportunity for Scottish families - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Opportunity for Scottish families - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: The Muscle Help Foundation is happy to announce that the charity's 2012 flagship Games Inspired Muscle Dreams programme is now live with the online nomination process officially open! This compelling project offers a once-in-a-lifetime opportunity for children and young people from across the UK with Muscular Dystrophy to witness the power and inspiration of the 2012 Paralympic Games.

Friday 25 May 2012

Action Duchenne trustee and supporter Mary Down becomes an Olympic torchbearer - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Action Duchenne trustee and supporter Mary Down becomes an Olympic torchbearer - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: On Friday 25th May, Mary Down, Action Duchenne trustee, supporter and grandmother will be carrying the Olympic Torch in Cardiff at about 17:40.�

Mary, who will be keeping her torch said that it is her ambition that all her grandchildren, including Seth will be able to bring out the torch in years to come when the Olympics returns to the UK.

Mary's grandson Seth has Duchenne Muscular Dystrophy and she was nominated by her son John for the amazing charity work she does.

PPMD endorses Senate version of Prescription Drug User Fee Act (PDUFA) after patient voice strengthened - PPMD Community

PPMD endorses Senate version of Prescription Drug User Fee Act (PDUFA) after patient voice strengthened - PPMD Community: As the U.S. Senate continues debate on legislation that seeks to accelerate delivery of safe and effective therapies to patients and to increase the patient voice as part of the therapy review process, PPMD�is pleased to offer our enthusiastic endorsement of the legislation.

Over the past several months, PPMD�has fought to include several key provisions included in the Senate version of the Food and Drug Administration Safety and Innovation Act, commonly referred to as the Prescription Drug User Fee Act or PDUFA.�A number of similar provisions are also included in a House bill expected to be voted on by that chamber next week.

The provisions included in the Senate user fee bill will help expand the patient voice when key decisions are being made, an issue of grave importance to our community and to the larger rare disease community.�The provisions will also provide the FDA with additional tools to more quickly review potential therapies for life-threatening conditions, including Duchenne.

Thursday 24 May 2012

Action Duchenne Scottish Parliament Lobby

Busy gathering my thoughts in preparation for the Action Duchenne 2012 lobby of the Scottish Parliament in a couple of weeks.

Points to be presented this year include:

- Provision of DEXA Scan services - section only was offered again but we have opted for a fully body scan to ensure full and properly comparable results are recorded. Note: partial scan results received at our last visit were not comparable with those from the full body scan at Logan's first appointment and so we will be insisting on full body scans from now on. How widespread is the offering of partial scans and are parent aware of their shortcomings?
- Relative scarcity of information and advice received from UK teams when compared to that provided to a friend in the US. DMD is a devastating and progressive disease and it is difficult enough just dealing with it, never mind having to constantly push for care, information and advice including scans, supplements, the use of antioxidants and so on.

Lots to do . . . .

Wednesday 23 May 2012

Super League’s Supermen feature Andy Johnson and the Joining Jack campaign

Super League’s Supermen feature Andy Johnson and the Joining Jack campaign - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Former rugby league player Andy Johnson and father to Jack aged 4, is to feature in an hour long feature to be aired this week on Sky Sports. Andy, who played for Salford City Reds, Castleford Tigers, London Broncos, Huddersfield Giants and Wigan Warriors in the European Super League has set up Joining Jack, a charity to help raise awareness of Duchenne and to raise funds to help find a cure. Filmed just last week, the programme Super League’s Supermen will broadcast on Sky Sports 4 on Thursday 24 May, at 7pm.

Action Duchenne donates �225,000 to Halo Therapeutics

Action Duchenne donates �225,000 to Halo Therapeutics - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Action Duchenne is proud to announce that we have agreed to support Halo Theapeutics in their quest to develop HT-100, the company’s proprietary formulation of halofuginone, a derivative of a compound found in a plant used in Chinese herbal medicine. HT-100 has great potential and may be able to halt or even reverse fibrosis, which is the scarring that occurs in muscles of Duchenne boys, inhibiting inflammation that leads to muscle damage, and promoting health muscle regeneration.

Friday 18 May 2012

Action Duchenne wins Good Cause Award!! - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Action Duchenne wins Good Cause Award!! - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Action Duchenne receives Good Cause Award from Bettys Tea Room, York. The Good Cause Award is a community grant of �500 awarded on the basis of supporting local families. We would like to thank the team at Bettys for their fantastic support.

Tuesday 15 May 2012

Study identifies how skeletal muscle stem cells respond to muscle injury

Study identifies how skeletal muscle stem cells respond to muscle injury: A study conducted by Children's Hospital & Research Center Oakland scientists identifies how skeletal muscle stem cells respond to muscle injury and may be stimulated to improve muscle repair in Duchenne Muscular Dystrophy, a severe inherited disease of muscle that causes weakness, disability and, ultimately, heart and respiratory failure.

Tuesday 8 May 2012

U of M researchers develop new muscular dystrophy treatment approach using human stem cells | Science Codex

U of M researchers develop new muscular dystrophy treatment approach using human stem cells | Science Codex: The research, published today in Cell Stem Cell, outlines the strategy for the development of a rapidly dividing population of skeletal myogenic progenitor cells (muscle-forming cells) derived from induced pluripotent (iPS) cells. iPS cells have all of the potential of embryonic stem (ES) cells, but are derived by reprogramming skin cells. They can be patient-specific, which renders them unlikely to be rejected, and do not involve the destruction of embryos.

This is the first time that human stem cells have been shown to be effective in the treatment of muscular dystrophy.

PPMD Holds Landmark Meeting with FDA Leaders to Address Trial Design, Endpoint,... -- HACKENSACK, N.J., May 3, 2012 /PRNewswire-USNewswire/ --

PPMD Holds Landmark Meeting with FDA Leaders to Address Trial Design, Endpoint,... -- HACKENSACK, N.J., May 3, 2012 /PRNewswire-USNewswire/ --: "The recent PPMD-FDA leadership meeting provides a solid foundation upon which we can build to develop and implement the tools and processes necessary to expedite the FDA's review of potential treatments and accelerate the delivery of safe and effective treatments to our patients," Furlong said.
"FDA leaders provided valuable information and perspectives on trial design and surrogate endpoint acceptance, two critically important issues, and expressed a desire to continue the discussion with industry leaders. PPMD looks forward to working with FDA to convene a larger roundtable discussion and other follow-up activities on these issues in the coming months, and will work with all patients and Duchenne stakeholders to ensure the community's voice is well-represented at these events."

Results from DuchenneConnect Study - PPMD Webinar Follow up - PPMD Community

Results from DuchenneConnect Study - PPMD Webinar Follow up - PPMD Community: Please see the list of Q&A's following the webinar video below, and�let me know if you have any additional follow up questions!

End of season Score4Duchenne Support - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

End of season Score4Duchenne Support - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Some of you may recall that Dundee FC Supporters Society kindly helped to support our Score4Duchenne appeal on Boxing Day.Today is the final day of the season and to celebrate and thank everyone involved with Dundee FC Supporters Society Community Project Action Duchenne will join other charities today at Dens Park.

At half time each of the charities from throughout the season will go onto the pitch bearing their own flag, kindly sponsored by Dundee FC, led by the Fintry Pipe Band to visually show the many good causes hosted this season and raise further awareness for the charities.

Tuesday 1 May 2012

MARTIN BASHIR, the TV journalist, explains why he’s backing the search for a cure for Duchenne muscular dystrophy.

Martin has become patron of Action Duchenne, the UK muscular dystrophy charity which campaigns for a cure and improved treatments for the cruel muscle-wasting condition which claimed the life of his brother Tommy whom he describes as “the most influential person in my life”.