Aims

Aim 1: Collect Data

Record at enrollment:

  • clinically relevant baseline characteristics and 
  • serum neurofilament light (NfL) measurements

Record at enrollment and each followup:

  • validated longitudinal measures of disease progression including 
    • ALSFRS-R and disease staging, 
    • respiratory vital capacity, 
    • speaking rate, and 
    • 10 meter walk test

Aim 2: Record Disease Milestones

Record disease milestones based upon 

  • patient-reported outcomes, 
  • durable medical equipment orders in the electronic health record, and 
  • other events such as dates of gastrostomy, hospitalizations, and death

Aim 3: Model Time-to-Events

Model time to events using baseline characteristics as well as retrospective and prospective longitudinal data

Additionally...

Datasets will be made available to academic and industry investigators to advance ALS care, drug development, and outcomes research. 

We will expand existing engagement from industry and other stakeholders to include an advisory panel of people living with ALS and will encourage opportunities for data dissemination through these existing relationships and publications.