Alstrom Syndrome

Positive Collaboration – Alström Syndrome UK

In Advocacy, Orphan Drug Congress, Partnering & Investment, Uncategorized by Freya Smale

Kerry Leeson-Beevers, National Development Manager at Alström Syndrome UK joined us at World Orphan Drug Congress Europe 2015 to provide insight into the role how positive collaboration has boosted their work.

Alström Syndrome is an ultra rare genetic condition with only 71 cases diagnosed in the UK. This autosomal recessive condition is extremely complex and life limiting.

Alström Syndrome UK was established in 1998 by 2 parents in Devon. Collaboration from specialists from Torbay Hospital helped to raise awareness as the group began to see more patients from elsewhere in the UK (voluntarily). Birmingham Children’s Hospital and Applied to the National Specialised Commissioning Group for specialised multi-disciplinary clinics soon became involved. ASUK are now equal partners in the delivery of the highly specialised service to patients with Alström Syndrome, funded by NHS England.

Use of the Compact –“Compact Voice works to ensure that strong, effective partnerships are at the heart of all relationships between the voluntary sector and government –locally and nationally”

Download this presentation to find about more about Alström Syndrome UK and how positive collaboration has enabled them to achieve:

  • Rare Disease Centres at BCH & QEH
  • Research Projects into Cardiac Investigations, Lifestyle Study and Potential Phase 2, Single-arm, Open Label Safety and Tolerability Study
  • New ASUK Projects including a Transition Project with £69,570 in funding received from BBC Children in Need
  • Opportunity to expand our Asian Mentoring Scheme
  • Patient Organisations – £150,000 secured funding

Get your copy of this presentation here >

For more information about the World Orphan Drug Congress Europe, download the brochure >

WODC EU 2016