LINK TO ONE OF OUR 52 MEMBER COUNTRIES FOR LOCAL SUPPORT, ADVICE AND NEWS

view map
close
  • Finland
  • Norway
  • Sweden
  • Estonia
  • Russian Federation
  • Latvia
  • Denmark
  • Lithuania
  • United Kingdom
  • Netherlands
  • Ireland
  • Poland
  • Germany
  • Belgium
  • Czech Republic
  • Ukraine
  • Slovakia
  • Luxembourg
  • Moldova, Republic of
  • Austria
  • France
  • Switzerland
  • Romania
  • Slovenia
  • Croatia
  • Serbia
  • Bosnia & Herzegovina
  • Bulgaria
  • Italy
  • Macedonia, the Former Yugoslav Republic of
  • Spain
  • Greece
  • Israel

Global Patient Registry For NGD

14 January 2019

The International Gaucher Alliance (IGA) are today pleased to announce the launch of Phase 1 of the development of a Global Patient Registry for neuronopathic Gaucher Disease (nGD) in order to increase disease knowledge and understanding, but also to develop new outcome measures and support emerging drugs/treatments in the pipeline.

Phase 1 of the project will take 16 weeks and will be led by the IGA and supported by the team from Elin Haf-Davies, Aparito. The project is being supported by a number of pharmaceutical companies interested in nGD including: Pfizer, OxyranePrevail Therapeutics and Sanofi Genzyme.

The project will involve 5 key areas:
1. Key opinion leader input
2. Patient engagement
3. Stakeholder steering group
4. Technical solution design and architecture
5. Global patient identification and feasibility/scoping exercise

Tanya Collin-Histed, CEO of the IGA says: “We are extremely excited about this project and with a number of companies interested in the development of new therapies for nGD the development of global patient registry, led by the patients will be an important tool to support a better understanding of the natural history of the disease, correlate global phenotypes and genotypes, validate new endpoints/ outcomes and to generate a date source that can be used for both Regulatory and Health Technology Assessments evaluation of emerging drugs for neuronopathic Gaucher disease”.

Dr Elin Haf Davies, CEO of Aparito added: "It is truly wonderful to be part of this patient driven initiative. Combining innovative approaches and global collaborative efforts in this way, with early dialogue among all the players will be sure to help move the needle significantly in this space”.

The IGA will share further updates on this project in due course.