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March 2010

A blooming great time to plant a winning sunflower for Action Duchenne ‘Seeds of Hope’ Appeal

Action Duchenne launches nationwide appeal to get children to grow sunflowers to raise funds for charity

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Looks Good, Tastes Great, gets great reviews

Carol Muskoron, Associate editor at www.allaboutyou.com, the online home of Good Housekeeping, House Beautiful, Prima, SHE, Country Living and Coast magazine reviews

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PTC answers questions on the cancelled Ataluren Trial

Nick Catlin from Action Duchenne attended a conference call organised by PPMD in the USA with PTC therapuetics. Here are his notes from the meeting.

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PTC Therapuetics have suspended the current Ataluren Trial for Duchenne

We have received this press release from PTC therapuetics concerning the Ataluren clinical trials

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February 2010

Report of the Parent Project International research Conference in Rome

Karl Bettelheim reports on the international research conference in Italy in February

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Action Duchenne launches new Cook Book “Looks Good, Tastes Great – Jewish Culinary Favourites”

Charity launches second cook book after success of ‘Caledonian Kitchen’ with all proceeds to support research

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Edward Ackroyd stars in new film directed by David Jason

Young Edward and his Mum Heidy are interviewed by GMTV

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Outstanding charity fundraiser Carl Tilson bids for Pride Of Manchester Awards 2009 - 2010

Manchester born, fund raiser and tireless champion for Duchenne Muscular Dystrophy in the running for Mancunion of the Year

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January 2010

Sunday People highlights Carl's campaign

Action Duchenne campaigner Carl Tilson, who is living with Duchenne Muscular Dystrophy, features in an article in the Sunday People supplement, Take it Easy

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Exciting new clinical trials to develop treatment for Duchenne Muscular Dystrophy - Press Release

Leading pharmaceutical development company for serious diseases and medical conditions undertakes first steps in bringing new treatment for DMD to market

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Exciting new clinical trials to develop treatment for Duchenne Muscular Dystrophy

Leading pharmaceutical development company for serious diseases and medical conditions undertakes first steps in bringing new treatment for DMD to market

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Initial results from systemic treatment with AVI-4658 positive

Positive RNA and Protein Signals in first cohorts (groups) analysed

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December 2009

Action Duchenne applauds publication of internationally agreed Standards of Care for the treatment of Duchenne Muscular Dystrophy

First step is taken towards bringing an end to the current ‘postcode lottery’ of care for those living with Duchenne

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TREAT-NMD Advisory Committee for Therapeutics is launched to guide best potential new therapies for the treatment of neuro-muscular diseases to clinical trials

Applications invited for comprehensive appraisals of new therapies by 15 December 2009

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November 2009

Action Duchenne and Duchenne Ireland funds further research for exon skipping therapies

Recent successful trials of RNA-based drug provide foundations for new therapies for more patients with Duchenne Muscular Dystrophy

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Swine flu advice from Treat NMD

Professor Hanns Lochmüller, Professor Volker Straub, Professor Kate Bushby

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Global excitement about potential treatments for Duchenne

Dr. Matthew Wood reports on recent developments in exon skipping trials from the Oligonucleotide Therapeutic Society meeting held in Fukuoka, Japan in November.

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Halloween Ball raises over £40k for Action Duchenne charity

Successful fundraising continues to support funding for research into Duchenne Muscular Dystrophy

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Action Duchenne funds appointment of new TREAT-Duchenne Coordinator

New role to help promote Internationally agreed Standards of Care for Duchenne Muscular Dystrophy

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October 2009

7th Annual Duchenne International Conference, most successful yet

Record attendance at Duchenne Conference

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AVI BioPharma Presents Updated Safety Data from Ongoing Systemic Trial of AVI-4658 at 7th Annual Action Duchenne International Conference

Data shosw PMO well tolerated in penultimate dose cohort (10mg/kg)

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AVI BioPharma to Present Safety Update from Ongoing Systemic Trial of AVI-4658 at 7th Annual Action Duchenne International Conference

Action Duchenne welcomes presentation of latest trial findings at annual conference

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Exon Skipping Drug Prevents Muscle Wasting and Maintains Muscle Function in Severely Affected, Dystrophin Deficient Mice

New Publication in Molecular Therapy Outlines Dramatic Effects in Animals Treated with Splice Switching PPMO, Demonstrates Promise for Treatment of Duchenne Muscular Dystrophy

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Keith Harris and Orville Support Action Duchenne Fundraising Event

Bicester Family organise fantastic charity auction event

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Personalised medicine: patient groups call on regulators to set approval route

from Science Business by Nuala Moran

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Prosensa and GlaxoSmithKline form alliance to fight Duchenne Muscular Dystrophy

Leiden and London, October 13 2009

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Sam joins MDEX exon skipping trial

Danny Smith Sams Dad reports on the latest AVI/MDEX trial

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TREAT-NMD, EMEA and patient group workshop on Antisense Oligonucleotide therapies in Duchenne muscular dystrophy (DMD) signals collaborative approach to the issues of personalised medicine

Action Duchenne sees positive step forward to paving way for individual genomic (designer) drugs to treat Duchenne

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September 2009

AVI BioPharma Phase 1 Proof of Concept and Safety Data for AVI-4658 in Duchenne Muscular Dystrophy Featured in Lancet Neurology

AVI-4658 Demonstrates Compelling Proof of Concept and Safety Profile for Pediatric Patients with DMD; Findings Support Progression to Current Systemic Use Trial

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August 2009

The Paper Mill at Apsley supports Action Duchenne with Carnival and Beer Festival over Bank Holiday Weekend

National charity for Duchenne Muscular Dystrophy to benefit from bank holiday family fun

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Action Duchenne supports Walton report findings and renews calls for ‘Centres of Excellence’ approach to treatment of muscular dystrophy

Walton Report confirms ‘postcode lottery’ to life expectancy for patients living with muscular dystrophy

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V Inspire Awards

nominate a genius volunteer

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Action Duchenne Replies to Health Minister

Nick Catlin our CEO replies to Gillian Merron at the Department of Heatlh

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July 2009

Patron Helena Bonham Carter supports Action Duchenne in its calls for the Government to follow recommendations of the House of Lords Genomic Medicine Report

Action Duchenne renews calls for ring fenced funding and more Centres of Excellence for the treatment of Duchenne Muscular Dystrophy in light of Government report

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AVI BioPharma and Action Duchenne Team Up to Support Advancement of PMO-based Therapeutics for Treatment of Duchenne Muscular Dystrophy

Action Duchenne provides $1.2m to support continuing development of drugs to treat Duchenne Muscular Dystrophy

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Action Duchenne supports bid for Pride of Britain Nomination by Carl ‘Action’ Tilson

Manchester terminally ill fund raiser bids for nomination to raise awareness of little known disease that is the biggest genetic childhood killer

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Genomics Medicine Campaign

Action Duchenne launches campaign to ask the Government to publish White paper

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Action Duchenne supports findings of Government Report on Genomic Medicine and renews calls for ring fenced funding and more Centres of Excellence

Single-gene disorders must not be left behind as new Genomic Medicines start to address more complex diseases

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Swine Flu and Duchenne

How to deal with Swine Flu outbreak

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June 2009

Action Duchenne lobbies Northern Ireland Assembly for Centres of Excellence and ‘Best Practice’ standards of medical care for all living with Duchenne Muscular Dystrophy

Parents and boys living with Duchenne march on Stormont to raise awareness of heartbreaking disease and demand an end to sub-standard care in Northern Ireland

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Action Duchenne lobbies Government for ‘Best Practice’ standards of medical care for all and for funding to develop a cure for Duchenne Muscular Dystrophy

Parents and boys living with Duchenne march on Downing Street to raise awareness of heartbreaking disease and demand an end to Postcode Lottery for medical care

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Action Duchenne lobbies Welsh Assembly for Centres of Excellence and ‘Best Practice’ standards of medical care for all living with Duchenne Muscular Dystrophy

Parents and boys living with Duchenne march on the Senedd to raise awareness of heartbreaking disease and demand an end to sub-standard medical care in Wales

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Action Duchenne calls for an end to the scandalous Postcode Lottery of treatment for boys and young men living with Duchenne Muscular Dystrophy

Standards of Care Survey shows that over a third of all parents of children living with Duchenne feel that their child receives sub-standard medical care

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Action Duchenne lobbies Scottish Parliament for Centres of Excellence and ‘Best Practice’ standards of medical care for all living with Duchenne Muscular Dystrophy

Parents and boys living with Duchenne march on Holyrood to raise awareness of heartbreaking disease and demand an end to sub-standard care in Scotland

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May 2009

World-renowned cellist Julian Lloyd Webber pledges support for Action Duchenne

Famous musician becomes latest patron for Action Duchenne, the Charity that supports families and those living with Duchenne

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Action Duchenne combines social networking with e-commerce for innovative new website

Action Duchenne builds online interactive community to help and support families living with Duchenne Muscular Dystrophy

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Race Against Time Campaign

Lets get lobbying

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Exon Skipping Review April 2009

from Günter Scheuerbrandt

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Action Duchenne launches ‘Life Thru a Lens’ photography project to raise awareness of Duchenne Muscular Dystrophy

Innovative photography project aims to show what living life with Duchenne is like by those whose lives are affected

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March 2009

Action Duchenne launches Celebrity Scottish Recipe Book - The Caledonian Kitchen

Scottish recipe book featuring favourite recipes from over forty celebrities and chefs from forty top Scottish restaurants raises funds for Duchenne Muscular Dystrophy charity

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February 2009

Action Duchenne announces new patent registry with Duchenne Ireland

Action Duchenne announces new patent registry with Duchenne Ireland

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St Valentine’s Day 10K Love Runs to be held across the UK for Action Duchenne

Muscular Dystrophy Charity holds 7th annual nationwide series of ‘Love Runs’ to raise money for research

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January 2009

AVI publishes latest Exon skipping clinical trial results

Latest drug trial shows potential RNA therapeutic treatment for Duchenne Muscular Dystrophy, says Action Duchenne

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