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Voxelotor Treatment Providing Transfusion Independence for Patient With Combined Sickle Cell Disease and Lipopolysaccharide-Responsive and Beige-Like Anchor (LRBA) Deficiency

We present a 22-year-old female with transfusion-dependent anemia due to sickle cell disease (SCD) with lipopolysaccharide-responsive and beige-like anchor protein (LRBA) deficiency whose treatment frequency was moderated with voxelotor (Oxbryta®). The patient was transfusion dependent, initially th...

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Detalles Bibliográficos
Autores principales:	Larkin, Kelsey M, Sharma, Archana, Castro, Liz, Drachtman, Richard
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Cureus 2023
Materias:	Pediatrics
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10651152/ https://www.ncbi.nlm.nih.gov/pubmed/38021883 http://dx.doi.org/10.7759/cureus.47144

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