Inspiratory muscle conditioning exercise and diaphragm gene therapy in Pompe disease: Clinical evidence of respiratory plasticity
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- 作者：Barbara K. Smitha ; b ; bksmith@phhp.ufl.edu" class="auth_mail" title="E-mail the corresponding author ; A. Daniel Martina ; Lee Ann Lawsonb ; Valerie Vernotc ; Jordan Marcuse ; Saleem Islamd ; Nadeem Shafif ; Manuela Cortib ; Shelley W. Collinsb ; Barry J. Byrneb
- 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
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Children with Pompe disease and ventilatory insufficiency received AAV1-CMG-GAA.
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Exercise alone did not change respiratory muscle function in any subjects.
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In less-affected children, dynamic respiratory function improved after gene therapy.