Limitations
AUTOIMMUNE DISORDERS, DISEASE, RARE DISEASE
Inherent to all rare disease research is the significant challenge to recruit a cohort of rigorously clinically characterized and monitored patients coupled to biospecimen collection and uniform treatment protocols. To date only the MGTX study fulfills all these requirements, and we took advantage of the biospecimen bank to perform a discovery study, which will require validation. The value of metabolomics and lipidomic analysis as an unbiased assessment for biomarker discovery and characterization of disease mechanisms cannot be questioned. Since the goal was to find lipids and metabolites that are treatment-predictive specific to MG patients, determining a profile specific to the disease itself is not relevant. In fact, we suspect the steroid-treatment response will be shared by other autoimmune disorders. There are numerous factors impacting the metabolome which are impossible to fully account for in any investigation. It is possible that improved strength will lead to increased activity [