Scleroderma remains a challenging disease with no effective therapy. Obstacles to development of effective therapies include the complex, multifactorial pathogenesis, lack of clear etiologic agent, uncertain role of genetic risks; patient-to-patient variability in clinical manifestations and disease outcomes; variable rates of disease progression in different organs in the same individual; lack of validated biomarkers, and lack of consensus regarding optimal clinical trial design. Each of these obstacles is being addressed by current research, and significant progress is being achieved. A large number of plausible drug targets have been identified and are being validated in preclinical models. Innovative clinical trial designs and increasingly more robust outcome measures allow more effective drug testing. Molecular omics approaches further facilitate patient stratification, and matching drugs to patients in an increasingly personalized precision approach to management.
Disclosure of Interest None declared