Presymptomatic IRB FAQ

What patient data is being collected through this observational study?
  • ALSFRSr, monthly 
  • ALS MOTOR exam, every 3 months 
  • LFTs monthly for the first 3 months on the drug, then every 3 months thereafter to ensure safety in taking riluzole
  • The results of a patient’s genetic testing, whether done before the study or as part of it, will be reviewed and confirmed by Synapticure’s Genetic Counselors so that the patient may enroll in the study
  • Additional tests may be added to this study in the future
How often will the data be analyzed?


How will this study’s inclusion in medical records impact patients? Is this part of the informed consent process?
  • This is described in the informed consent. In short, this data cannot be shared with health insurance companies, but life insurance and disability insurance may have access. 
  • If a patient has previously tested positive for a pathogenic ALS variant, your life insurance and disability insurance may already have that information.
What are the estimated costs?
  • These will be standard physician visits, so the patient will pay their insurance’s copay (most fall between $0-40/visit)
  • Medication is about $13/month (source)
  • For patients unable to afford the cost of the visits, Synapticure has multiple avenues available to help address costs
How many participants can enroll?

The initial IRB can accommodate up to 150 patients, but we may exceed that. If we see it trending in that direction, we will go back to the IRB with an amendment.

Will the data be shared?

All data will be shared with the patient. This is explicit in the ICF, which also mentions that this data may have the potential to cause stress, anxiety, etc., which we added for full transparency to ensure full respect for persons and their autonomy.