Patient-Reported Outcomes in Autosomal Dominant Osteopetrosis: Findings from the Osteopetrosis Registry Study.
Lynda E PolgreenEva Villa-LopezLiz ChenZiyue LiuAmy KatzCorinne Parks-SchenckMarian HartErik A ImelMichael J EconsPublished in: The Journal of clinical endocrinology and metabolism (2024)
Data from this registry demonstrate the broad spectrum of ADO disease severity and high impact on health-related quality of life in adults with ADO.