Fully Fund the first treatment for Duchenne Muscular Dystrophy

UPDATE: 15/04/16

We won! Thank you for signing the petition Fully Fund the first treatment for Duchenne Muscular Dystrophy. Due to your fantastic support today NICE announce funding for the first genetic treatment for Duchenne in the UK.

Read more here: http://www.actionduchenne.org/nice-recommend-translarna-for-treating-nmdmd/

NC
Nick Catlin
Campaign created by
Nick Catlin
29,220
of 30,000 signatures

To: NHS England

Families have been waiting a long time for new treatments to come on stream and everyone in the Duchenne community urges the NHS in England, Scotland, Wales and Northern Ireland to fully fund the costs of Translarna and support its immediate delivery to patients.

Why is this important?

Translarna is the first novel drug to be granted market approval for Duchenne Muscular Dystrophy (DMD) that treats the underlying causes of the condition. DMD is a life limiting condition for which there is no cure. The severe and progressive muscle wasting leaves young adults reliant on wheelchairs, ventilators and at severe risk of early heart failure. The US company PTC Therapeutics have been granted marketing authorisation in the European Union under the trade name Translarna for the treatment of Duchenne for a sub group of patients aged five years and older who are walking and have a single point variation in their dystrophin gene.
This is a crucial landmark in treating Duchenne. Translarna is expected to be the first of other personalised medicines that are currently in clinical trial and could also become available to treat more young people with DMD.
It is now imperative that all children who are identified with these specific Duchenne gene mutations have long term access to this novel therapy.

Category: Health

Reasons for signing

  • Elaine S. 2016-07-28 12:34:36 +0100
    In loving memory of my two nephews who fought this despicable disease and for every other young boy out there xx
  • Zoe B. 2016-06-20 16:37:17 +0100
    This is a vile disease. It needs to be treated and to be prevented by selective breeding.
  • Miriam S. 2016-05-05 19:23:44 +0100
    My son was diagnosed with this, unbelievable the test results were mixed up. That was in 1986 and I never forget what it was like for 2 whole weeks to think my son had this. My boy is 31 and has 2 lovely kids. I am so lucky. So come on and do the right thing for these kids. It's tragic.

Updates

4 months ago
Success badge small

Petition is successful with 28,958 signatures

over 1 year ago

20,000 signatures reached

over 1 year ago

Please note change of date: All UK Duchenne and Muscular Dystrophy organisations are backing a lobby of parliament on January 14th 2015 to deliver this petition to the Prime Minister. Meet at Portcullis House opposite Big Ben from 10.30am. Contact your MP and agree to meet them during the morning to brief them about the campaign. Lets see if we can get past 20000

over 1 year ago

Thank you to everyone who has helped to get us to #10000. We need to keep spreading the word. NHS England meet on the 8th December to discuss funding Translarna. We will send the petition electronically to Sir Nick Partridge,Chair, Clinical Priorities Advisory Group, NHS England,PO Box 16738 Redditch B97 9PT before the meeting of the CPAG.
The final decision is with the NHS Board in early February 2015. We will be organising a lobby of the Department of Health in Whitehall and take hard copies to No 10 Downing Street on Wednesday 4th February 2015 before the final NHS Board meeting. So we need all the signatures that everyone can organise. More details of lobby event to follow. Please read Rachel and Bernies Story about their son Ben: http://dmd79.blogspot.co.uk/2014/12/funding-translarna-means-we-still-have.html

over 1 year ago

10,000 signatures reached

over 1 year ago

5,000 signatures reached

over 1 year ago

1,000 signatures reached

over 1 year ago

500 signatures reached

over 1 year ago

Once you have signed the petition don't forget to ask your MP, MSP, AM's MLA's MEP's to sign and publicise You can use: https://www.writetothem.com/ to find your representative and then send them this link https://you.38degrees.org.uk/p/translarna

over 1 year ago

100 signatures reached

over 1 year ago

50 signatures reached

over 1 year ago

25 signatures reached

over 1 year ago

10 signatures reached