Late-Onset GM2 gangliosidosis (LOTS) is a rare and progressive neurological genetic disorder that has a devastating impact on those it affects. There is no cure and treatment for the disease currently focuses on managing symptoms.

Digital remote patient monitoring technology has been identified as having the potential to aid the drug development process. By developing new digital endpoints for clinical trials it will be possible to deliver healthcare and treatments tailored to the specific needs of patients. Innovative digital approaches for rare diseases can provide great opportunities for drug development, but they need to be suitable for the patients that will use them to ensure that sufficient data sets are generated.

In this white paper we summarise this innovative approach for a rare lysosomal storage disease (LSD) to show:

  • The potential application of remote patient monitoring technology for an LSD in adults.
  • The accessibility of the Aparito Atom5™ platform for patients with neurodegenerative diseases such as LOTS.
  • The importance of collecting real-world evidence for a rare disease to help develop a greater knowledge of the disease and its impact on the individual.
  • That LOTS patients have many unmet clinical needs that need to be addressed with emerging therapies.