Drug development for neuronopathic Gaucher disease (nGD) is compounded by its heterogeneity and the lack of explicit clinical hallmarks which would be pivotal for differentiating between its subtypes. 

Type 3 nGD is a disease for which there is no cure, and currently, no medicines for its treatment exist. Remote patient monitoring, through the deployment of a wearable device, paired with a smartphone app, can potentially advance the drug development process by providing real-world data, and real-world evidence for the disease.

In this whitepaper the following will be explored:

  • The unmet clinical needs of nGD patients 
  • nGD data aggregation via the Atom5™ remote patient monitoring platform, in the UK Gaucher Association and Aparito clinical study
  • Clinical differences observed between type 1 Gaucher disease and type 3 nGD patients via Atom5™
  • Applicability of Atom5™ for nGD patients, and clinicians