Professor Sir Malcolm Grant OBE            Ed Smith, CBE, FCA Ciaran Devane Margaret Casely-Hayford Dame Moira Gibb Lord Victor Adebowale Professor Sir John Burn Noel Gordon David Roberts Simon Stevens Professor Sir Bruce Keogh Jane Cummings Dame Barbara Hakin Paul Baumann Tim Kelsey Karen Wheeler

Six months ago we launched our campaign – and today we are delighted to announce our first significant victory in our Fight4Treatment.  The Health Service Journal (HSJ) had today reported that NHS England are to publish a policy that will allow patients with urgent need

Greg MulHolland MP led a debate in parliament yesterday (16 June 2015) to highlight failures access to medicines for rare diseases.  The debate was well-attended debate supported by MPs from all political parties.  Mr MulHolland highlighted Morquio Syndrome, Duchenne Muscular Dystrophy and Tuberous Sclerosis Complex. 

1 April, 2015 | By Shaun Lintern NHS England has been accused of misleading government ministers and labelled a ‘dysfunctional organisation’ over delays in drawing up a prescribing policy for drugs to treat rare diseases. Health charities and Liberal Democrat MP Greg Mulholland have also

Greg Mulholland MP has secured a parliamentary debate entitled ‘Access to drugs for ultra-rare diseases’! It’ll be next Tuesday, 16th June, 2.30pm-4pm, so a whole hour and a half of MPs raising their concerns directly with George Freeman.You can help by emailing and/or tweeting your

When Everolimus was licensed for the treatment of TSC none of us imagined that years later we would still be fighting for access.   Over the past 2 years, the TSA has been supporting the development of a service specification and prescribing policy in the

NHS England has now published the long-awaited urgent and critical need policy. If you or someone you care for has had an individual funding request rejected for access to Everolimus, and their need is urgent, the new policy should be of help to your doctor

When Everolimus was licensed for the treatment of TSC, no-one imagined that more than 2 years later we would be campaigning for access in the face of a near total failure in NHS funding in England.   Now, thanks to the many of you who

When Everolimus was licensed for the treatment of TSC more than 2 years ago, no-one imagined that years later we would still be fighting for access in the face of a near total failure in NHS funding in England.    Over the past year and

On 17th September, the Welsh Government wrote to NHS Chief Executives across Wales to inform them that an agreement had been reached with Novartis Pharmaceuticals UK Limited for the provision of Everolimus for TSC patients.    This two-year agreement is the first of it’s kind