“…In contrast to clinical trials, which by definition are limited to small patient numbers in rare diseases, rare disease registries afford the opportunity to amass real-world longitudinal data from larger cohorts, including children, pregnant women, asymptomatic patients, and patients with neuronopathic GD, over longer time periods, while collection of cross-sectional registry data allow the reconstruction of natural histories, which would otherwise not be evident in rare disorders [8]. A number of international registries for patients with GD exist, the largest being the International Collaborative Gaucher Group disease registry (ClinicalTrials.gov Identifier: NCT00358943), initiated by Genzyme in 1991 (now Sanofi), followed by the Gaucher Outcome Survey (GOS) disease registry, initiated in 2010 by Shire (now Takeda) after US Food and Drug Administration approval of velaglucerase alfa, and a drug-specific registry Taliglucerase Alfa Surveillance, for patients receiving treatment with taliglucerase alfa, initiated by Pfizer in 2013 (EUPAS4721 [9]), as well as national registries including the Spanish Gaucher Disease Registry, initiated in 1993 [10], the French Gaucher Disease Registry, initiated in 2009 [11], and the GAUCHERITE registry initiated in 2014 in the United Kingdom [12].…”