What kinds of questions might the CFR help answer?

Title: 
What kinds of questions might the CFR help answer?

There are many questions that may be answered through the CFR. For example:

  1. What is the global population of individuals with SLSMD disorders, and broken down by phenotype (Pearson syndrome, KSS, CPEO)?
  2. What is the natural history progression of SLSMD disorders?
  3. What are potential new pathways for treatments and cures?