dmd79.blogspot.com
NICE decision for use of Translarna - a criminal act | 79
http://dmd79.blogspot.com/2015/10/nice-decision-for-use-of-translarna.html
NICE decision for use of Translarna - a criminal act. Saturday, 17 October 2015. The decision this week from NICE in the UK to deny boys with Duchenne Muscular Dystrophy the use of the drug Translarna is criminal. This first use of a genetic therapy for Duchenne has been approved by the EMA. And other European countries have already agreed to fund the use of Translarna. In Scotland one young person, Cormac Fegan. Has been granted funding for Translarna. The current decision by NICE, to be further reviewe...
dmd79.blogspot.com
79 | 79
http://dmd79.blogspot.com/2014/11/79.html
Thursday, 27 November 2014. In the 1850's Guillaume-Benjamin-Amand Duchenne (de Boulogne) discovered Muscular Dystrophy. In fact, the original name of this disease was Duchenne Muscular Dystrophy, named in his honor. But it took until 1986, for the gene dystrophin to be cloned, until we had a real scientific basis for understanding the mechanisms underlying the severe muscle wasting first seen by Duchenne over 130 years before. From Dr. Annemieke Aartsma-Rus. At first it was hoped that the dystrophin gen...
dmd79.blogspot.com
Open letter to Sir Nick Partridge Chair of CPAG | 79
http://dmd79.blogspot.com/2014/12/open-letter-to-sir-nick-partridge-chair.html
Open letter to Sir Nick Partridge Chair of CPAG. Tuesday, 2 December 2014. Dear Sir Nick Partridge. So it is vital that the CPAG committee understand the need to fund Translarna and also other new medicines when they become licensed. Saul suggested that I set up a petition. Https:/ you.38degrees.org.uk/p/translarna. And I’m delighted to say that the Duchenne Community and many others (including the dog Elvis! Are supporting our call to fully fund this treatment. Families have been waiting a long time for...
dmd79.blogspot.com
Carmeseal-MD | 79
http://dmd79.blogspot.com/2014/12/carmeseal-md.html
Thursday, 11 December 2014. An early access programme was announced for Carmeseal-MD™ (Poloxamer 188, NF) recently ( http:/ www.phrixuspharmaceuticals.com/news.php. Pharmaceuticals announces European Access Program for CarmesealMD™ (P-188 NF) for. Patients with Duchenne muscular dystrophy". Treat NMD and UPPMD have published this very good guideline for families http:/ www.treat-nmd.eu/dmd/carmeseal/. So it might be the case here that patients can find clinicians that Phrixus are willing to offer Carmese...
dmd79.blogspot.com
Campaign for Translarna | 79
http://dmd79.blogspot.com/2014/11/campaign-for-translarna.html
Thursday, 27 November 2014. Translarna is the first novel drug to be granted market approval for Duchenne Muscular Dystrophy that treats the underlying causes of the condition. PTC Therapeutics. Have been granted marketing authorisation in the European Union under the trade name Translarna™ for the treatment of Duchenne patients aged five years and older. At Action Duchenne if they are not sure of their child's genetic diagnosis or if their child might benefit from this drug. We urgently need a united an...
dmd79.blogspot.com
Funding Translarna means we still have hope | 79
http://dmd79.blogspot.com/2014/12/funding-translarna-means-we-still-have.html
Funding Translarna means we still have hope. Monday, 1 December 2014. Was Rachel being a little paranoid when she said something was wrong? Ben wouldn’t do the things she had read about avidly both during pregnancy and after he was born. He missed milestones and we even moved house so that he wouldn’t have to go to the tiny village school – we thought he needed pushing more – so we moved and thought we’d solved a problem for our ‘laid back’ happy little boy. In 2013 an extension trial finally started aga...
SOCIAL ENGAGEMENT