Genetic basis and therapies for vascular anomalies

A Queisser, E Seront, LM Boon, M Vikkula - Circulation research, 2021 - Am Heart Assoc
Vascular and lymphatic malformations represent a challenge for clinicians. The identification
of inherited and somatic mutations in important signaling pathways, including the PI3K …

[HTML][HTML] Efficacy and safety of sirolimus in the treatment of vascular anomalies: a systematic review

C Freixo, V Ferreira, J Martins, R Almeida… - Journal of vascular …, 2020 - Elsevier
Objective The management of vascular anomalies is complex and requires a
multidisciplinary team with a combination of medical, surgical, and intervention treatments …

New and emerging targeted therapies for vascular malformations

A Van Damme, E Seront, V Dekeuleneer… - American journal of …, 2020 - Springer
Vascular malformations are inborn errors of vascular morphogenesis and consist of
localized networks of abnormal blood and/or lymphatic vessels with weak endothelial cell …

Generalized lymphatic anomaly and Gorham–Stout disease: overview and recent insights

M Ozeki, T Fukao - Advances in wound care, 2019 - liebertpub.com
Significance: Generalized lymphatic anomaly and Gorham–Stout disease are extremely rare
diseases with severe symptoms and poor prognosis. The etiology and clinical presentation …

Model-informed precision dosing of antibiotics in pediatric patients: a narrative review

A Abdulla, E Edwina, RB Flint, K Allegaert… - Frontiers in …, 2021 - frontiersin.org
Optimal pharmacotherapy in pediatric patients with suspected infections requires
understanding and integration of relevant data on the antibiotic, bacterial pathogen, and …

Sirolimus treatment in Sturge-Weber syndrome

AJ Sebold, AM Day, J Ewen, J Adamek, A Byars… - Pediatric …, 2021 - Elsevier
Abstract Background Sturge-Weber syndrome is a rare neurovascular disorder associated
with capillary malformation, seizures, cognitive impairments, and stroke-like episodes …

Eculizumab precision-dosing algorithm for thrombotic microangiopathy in children and young adults undergoing HSCT

K Mizuno, CE Dandoy, A Teusink-Cross… - Blood …, 2022 - ashpublications.org
Transplant-associated thrombotic microangiopathy (TA-TMA) is a fatal posttransplant
complication of hematopoietic stem cell transplantation. We recently reported that survival …

[HTML][HTML] GNTI-122: an autologous antigen-specific engineered Treg cell therapy for type 1 diabetes

GI Uenishi, M Repic, JY Yam, A Landuyt… - JCI insight, 2024 - ncbi.nlm.nih.gov
Tregs have the potential to establish long-term immune tolerance in patients recently
diagnosed with type 1 diabetes (T1D) by preserving β cell function. Adoptive transfer of …

Vascular malformations: An overview of their molecular pathways, detection of mutational profiles and subsequent targets for drug therapy

A Mansur, I Radovanovic - Frontiers in Neurology, 2023 - frontiersin.org
Vascular malformations are anomalies in vascular development that portend a significant
risk of hemorrhage, morbidity and mortality. Conventional treatments with surgery …

Tutorial on model selection and validation of model input into precision dosing software for model‐informed precision dosing

ZL Taylor, EA Poweleit, K Paice… - CPT …, 2023 - Wiley Online Library
There has been rising interest in using model‐informed precision dosing to provide
personalized medicine to patients at the bedside. This methodology utilizes population …