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Prasad, Suyash
142  results:
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1

SLC13A5 Deficiency Disorder: From Genetics to Gene Therapy:

Goodspeed, Kimberly ; Liu, Judy S. ; Nye, Kimberly L....
Genes.  13 (2022)  9 - p. 1655 , 2022
Link: https://doi.org/10.3390/..
 
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2

OP018: ASPIRO gene therapy trial in X-Linked Myotubular Myo..:

Shieh, Perry ; Kuntz, Nancy ; Dowling, James...
Genetics in Medicine.  24 (2022)  3 - p. S350 , 2022
Link: https://doi.org/10.1016/..
 
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3

Connect: Designing a first-in-human gene replacement therap..:

Leal-Pardinas, Fernanda ; Cunningham, Andy ; Okposo, Kome...
Molecular Genetics and Metabolism.  135 (2022)  2 - p. S73 , 2022
Link: https://doi.org/10.1016/..
 
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4

Gene Therapy: Novel Approaches to Targeting Monogenic Epile..:

Goodspeed, Kimberly ; Bailey, Rachel M. ; Prasad, Suyash...
Frontiers in Neurology.  13 (2022)  - p. , 2022
Link: https://doi.org/10.3389/..
 
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5

Use of the Children's Hospital of Philadelphia Infant Test ..:

Duong, Tina ; Harding, Gale ; Mannix, Sally...
Journal of Neuromuscular Diseases.  8 (2021)  1 - p. 63-77 , 2021
Link: https://doi.org/10.3233/..
 
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6

ASPIRO Gene Therapy Trial In X-Linked Myotubular Myopathy (..:

Shieh, Perry B. ; Kuntz, Nancy ; Smith, Barbara...
Neurology.  94 (2020)  15_supplement - p. , 2020
Link: https://doi.org/10.1212/..
 
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7

Pre-clinical safety and efficacy findings of AT845, a novel..:

Mavilio, Fulvio ; Cunningham, Justine ; Eggers, Michelle...
Molecular Genetics and Metabolism.  129 (2020)  2 - p. S106 , 2020
Link: https://doi.org/10.1016/..
 
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8

A Systematic Review of the Health Economics of Pompe Diseas:

Schoser, Benedikt ; Hahn, Andreas ; James, Emma...
PharmacoEconomics - Open.  3 (2019)  4 - p. 479-493 , 2019
Link: https://doi.org/10.1007/..
 
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9

Continued analysis of GAA mice treated with novel hybrid pr..:

Heffner, Garrett ; James, Lucy ; Chaivorapol, Christina...
Molecular Genetics and Metabolism.  126 (2019)  2 - p. S70 , 2019
Link: https://doi.org/10.1016/..
 
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10

Clinical program to evaluate safety, preliminary efficacy, ..:

Rico, Salvador ; Schoser, Benedikt ; Dimmock, David...
Molecular Genetics and Metabolism.  126 (2019)  2 - p. S123 , 2019
Link: https://doi.org/10.1016/..
 
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11

Disease burden of Crigler–Najjar syndrome: Systematic revie..:

Dhawan, Anil ; Lawlor, Michael W ; Mazariegos, George V...
Journal of Gastroenterology and Hepatology.  35 (2019)  4 - p. 530-543 , 2019
Link: https://doi.org/10.1111/..
 
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12

Mortality and respiratory support in X-linked myotubular my..:

Graham, Robert J ; Muntoni, Francesco ; Hughes, Imelda...
Archives of Disease in Childhood.  105 (2019)  4 - p. 332-338 , 2019
Link: https://doi.org/10.1136/..
 
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13

A multicenter, retrospective medical record review of X‐lin..:

Beggs, Alan H. ; Byrne, Barry J. ; De Chastonay, Sabine...
Muscle & Nerve.  57 (2017)  4 - p. 550-560 , 2017
Link: https://doi.org/10.1002/..
 
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14

The humanistic burden of Pompe disease: are there still unm..:

Schoser, Benedikt ; Bilder, Deborah A. ; Dimmock, David...
BMC Neurology.  17 (2017)  1 - p. , 2017
Link: https://doi.org/10.1186/..
 
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15

Skeletal Muscle Pathology in X-Linked Myotubular Myopathy: ..:

Lawlor, Michael W. ; Beggs, Alan H. ; Buj-Bello, Ana...
Journal of Neuropathology & Experimental Neurology.  75 (2016)  2 - p. 102-110 , 2016
Link: https://doi.org/10.1093/..
 
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