Trial Interviews to Explore Glycogen Storage Disease Type Ia Patient Experiences Following Gene Therapy

# Background Glycogen storage disease type Ia (GSDIa) is a rare, inherited, autosomal recessive deficiency of glucose-6-phosphatase (G6Pase), an enzyme necessary in glycogenolysis and gluconeogenesis. To maintain normal blood glucose levels and ensure survival, individuals living with GSDIa must fre...

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Principais autores:	Diane M. Turner-Bowker, Jessica Butler, Shayna Egan, David A. Weinstein, David F. Rodriguez-Buritica, Ayesha Ahmad, María-Luz Couce, Rebecca Riba-Wolman, John J. Mitchell, Christina Theodore-Oklota
格式:	Artigo
語言:	Inglês
出版:	Columbia Data Analytics, LLC 2026-02-01
叢編:	Journal of Health Economics and Outcomes Research
在線閱讀:	https://doi.org/10.36469/001c.155666
標簽:	添加標簽 沒有標簽, 成為第一個標記此記錄!