Earlier this year we joined forces with the Genetics Alliance and the Association for Glycogen Storage Disorders to petition the National Assembly for Wales to review the use of the exceptionality rule in determining whether a patient can access a treatment through the Individual Patient

We are delighted to announce a new award to support career development of young scientists.  The first Ann Hunt Career Development Award to will support a PhD student working with Professor Petrus de Vries, formerly in Cambridge and now at the University of Cape Town,

NHS England is currently failing to properly support patients with TSC and the dedicated NHS professionals who care for them. It is vitally important that we continue to campaign for implementation of the international TSC guidelines and for access to medicine. Whether you have

On the 15th November 2014 the TSA hosted another successful Family Annual Conference. With specialist workshops on Coping and Caring for those with TSC, Campaigning for fair access to treatment, Physiological Issues and TSC, Behavioural Challenges, Research and ‘Finding your way through the legal maze’

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

Prime Minister fails to recognise TSC patients in critical need 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

Exclusive: NHS England drug delay led to ‘absolutely preventable’ deaths 17 March, 2015 | By Shaun Lintern A two year delay in NHS England drawing up a prescribing policy for a licensed drug has led to at least two ‘absolutely preventable’ deaths and more than 30 adults and

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.