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

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

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作者：Barbara K. Smith^a ; ^b ; ^{bksmith@phhp.ufl.edu" class="auth_mail" title="E-mail the corresponding author} ; A. Daniel Martin^a ; Lee Ann Lawson^b ; Valerie Vernot^c ; Jordan Marcus^e ; Saleem Islam^d ; Nadeem Shafi^f ; Manuela Corti^b ; Shelley W. Collins^b ; Barry J. Byrne^b
关键词：ERT ; Enzyme replacement therapy ; GAA ; α-glucosidase ; IMST ; Inspiratory muscle strength training ; LC ; Load compensation ; MIP ; Maximal inspiratory pressure ; MRI ; Magnetic resonance imaging ; MV ; Mechanical ventilation
刊名：Experimental Neurology
出版年：2017
出版时间：January 2017
年：2017
卷：287
期：part_P2
页码：216-224
全文大小：709 K

文摘

•: Children with Pompe disease and ventilatory insufficiency received AAV1-CMG-GAA.
•: Exercise alone did not change respiratory muscle function in any subjects.
•: In less-affected children, dynamic respiratory function improved after gene therapy.

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