Campaign for Translarna Nick Catlin Thursday, 27 November 2014 No Comment

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.

This is a crucial landmark in treating Duchenne despite only being targeted at a small sub population of children that are currently experiencing the severe muscle wasting effects of this condition. Young poeple will have to have a single point mutation of the dystrophin gene in order for Translarna to have a chance of reading through the gene to produce a functional protein. Parents should check with the DMD Registry at Action Duchenne if they are not sure of their child's genetic diagnosis or if their child might benefit from this drug.

Right now NHS England are considering whether or not to pay for the costs of this drug to patients.
The Muscular Dystrophy Campaign are heading up a campaign to get Translarna properly rolled out and funded.

But should we all be doing much more?

This will be a vital test case with the NHS in England, Scotland, Wales and Northern Ireland to see if they will provide substantial funding and support to offer treatment to a small number of patients with rare diseases like Duchenne Muscular Dystrophy. There are other potential therapies coming downstream for Duchenne and my fear is that in the current climate of cut backs and austerity that Translarna will not get funded. If that is the case then this will massively jeopardise access to other treatments downstream.

In the US recently Duchenne families and organisations united in a fantastic campaign with the FDA to get them to shift support for faster access to the exon skipping drug at Sarepta .

We urgently need a united and vocal campaign from all UK Duchenne and Muscular Dystrophy Charities to win NHS funding for Translarna. We need to involve all parents, young people and families and mobilise them to contact our MP's and organise lobbies at the DoH.  Winning this campaign is a vital step forward for new and better medicines for all our children.
79 is hosted by Nick Catlin

Nick Catlin has a son Saul who has Duchenne Muscular Dystropy. He is a founding member and previous CEO of the Charity Action Duchenne. Nick now works with young people with Duchenne and their families at Decipha assessing for Special Education Needs and advising on reviewing Education Health and Care Plans.

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