日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Inspiratory muscle conditioning exercise and diaphragm gene therapy in Pompe disease: Clinical evidence of respiratory plasticity

庞贝病吸气肌调节训练和膈肌基因治疗:呼吸可塑性的临床证据

Smith, Barbara K; Martin, A Daniel; Lawson, Lee Ann; Vernot, Valerie; Marcus, Jordan; Islam, Saleem; Shafi, Nadeem; Corti, Manuela; Collins, Shelley W; Byrne, Barry J

Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease

经膈肌递送腺相关病毒介导的α-葡萄糖苷酶(rAAV1-CMV-hGAA)基因疗法治疗庞贝病患儿的安全性

Corti, Manuela; Liberati, Cristina; Smith, Barbara K; Lawson, Lee Ann; Tuna, Ibrahim S; Conlon, Thomas J; Coleman, Kirsten E; Islam, Saleem; Herzog, Roland W; Fuller, David D; Collins, Shelley W; Byrne, Barry J

Phase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomes.

腺相关病毒介导的α-葡萄糖苷酶基因疗法治疗庞贝氏症慢性呼吸衰竭的I/II期试验:初步安全性和通气结果

Smith Barbara K, Collins Shelley W, Conlon Thomas J, Mah Cathryn S, Lawson Lee Ann, Martin Anatole D, Fuller David D, Cleaver Brian D, Clément Nathalie, Phillips Dawn, Islam Saleem, Dobjia Nicole, Byrne Barry J

B-Cell depletion and immunomodulation before initiation of enzyme replacement therapy blocks the immune response to acid alpha-glucosidase in infantile-onset Pompe disease

在开始酶替代疗法之前进行B细胞清除和免疫调节,可阻断婴儿型庞贝病患者对酸性α-葡萄糖苷酶的免疫反应。

Elder, Melissa E; Nayak, Sushrusha; Collins, Shelley W; Lawson, Lee Ann; Kelley, Jeffry S; Herzog, Roland W; Modica, Renee F; Lew, Judy; Lawrence, Robert M; Byrne, Barry J

Pompe disease gene therapy

庞贝病基因治疗

Byrne, Barry J; Falk, Darin J; Pacak, Christina A; Nayak, Sushrusha; Herzog, Roland W; Elder, Melissa E; Collins, Shelley W; Conlon, Thomas J; Clement, Nathalie; Cleaver, Brian D; Cloutier, Denise A; Porvasnik, Stacy L; Islam, Saleem; Elmallah, Mai K; Martin, Anatole; Smith, Barbara K; Fuller, David D; Lawson, Lee Ann; Mah, Cathryn S