Rare Diseases Blogs
Worth the wait – The Goldacre review
Our thoughts on Professor Ben Goldacre’s “Better, broader, safer” report into health data.
Innovative medicines can bring hope to people with rare diseases – let’s get them to patients
England’s Action Plan for Rare Diseases contains important commitments to ensure that people with rare diseases get faster diagnosis, better care and timelier access to the treatments they desperately need.
By working together we can improve the lives of people with rare diseases
Many people are surprised to learn that rare diseases are actually comparatively common. A rare disease is defined as one that affects fewer than one in 2,000 people and it is reckoned that 300 million people worldwide have one. More than 6,000 rare diseases have been identified and about 72% of those have a genetic origin.
Moving beyond box-ticking and lip service – why patient involvement matters in a new era of ATMPs for rare diseases
Laurence Woollard is a patient advocate for rare diseases and a Patient and Public Voice (PPV) Partner on NHS England’s ATMP working group. For Rare Disease Day, his message is that meaningful patient involvement is essential to achieve greater access to potentially life-changing therapies for rare diseases.
Rare disease Hub
Rare diseases have a devastating impact on people’s lives: pharmaceutical companies here in the UK and around the world are working to change that – developing new medicines to give families hope for the future.
The UK Rare Diseases Framework has been published, a national vision of how the UK will improve the lives of those living with rare diseases. Industry wants to help fulfil its important priorities. Read the ABPI statement.