Doux J. Barriers and Opportunities: A View Across the Developmental Divide. Journal of Investigative Dermatology (2015) 135, 2143–2144.
Licholai G. Rare Dermatology: Orphan Disease Drug Development Opportunities and Challenges. Dermatology Summit (2019).
Hammill AM, et al., Sirolimus for the treatment of complicated vascular anomalies in children. Pediatric Blood and Cancer (2011).
Adams DM, et al., Efficacy and Safety of Sirolimus in the Treatment of Complicated Vascular Anomalies. Pediatrics (2016).
Canaud G, et al., A review of mechanisms of disease across PIK3CA-related disorders with vascular manifestations. Orphanet Journal of Rare Diseases (2021).
Carroll S, et. al., Annual prevalence estimation of lymphatic malformation with a cutaneous component: observational study of a national representative sample of physicians. Orphanet Journal of Rare Diseases (2022).