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Kuntz, N.
123
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1
AMFR dysfunction causes autosomal recessive spastic paraple..:
Deng, R
;
Medico-Salsench, E
;
Nikoncuk, A
...
https://discovery.ucl.ac.uk/id/eprint/10169693/1/s00401-023-02579-9.pdf. , 2023
Link:
https://discovery.ucl.ac..
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2
Safety and effectiveness of ataluren: comparison of results..:
Mercuri, E
;
Muntoni, F
;
Osorio, AN
...
info:eu-repo/semantics/altIdentifier/wos/WOS:000533276600004. , 2020
Link:
http://hdl.handle.net/24..
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3
Deflazacort vs prednisone treatment for Duchenne muscular d..:
McDonald, CM
;
Sajeev, G
;
Yao, Z
...
https://discovery.ucl.ac.uk/id/eprint/10110700/1/mus.26736.pdf. , 2020
Link:
https://discovery.ucl.ac..
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4
Serum biomarkers associated with baseline clinical severity..:
Dang, UJ
;
Ziemba, M
;
Clemens, PR
...
issn:0964-6906. , 2020
Link:
http://hdl.handle.net/11..
?
5
TCTEX1D1 is a genetic modifier of disease progression in Du..:
Spitali, P
;
Zaharieva, I
;
Bohringer, S
...
issn:1018-4813. , 2020
Link:
http://hdl.handle.net/11..
?
6
Spinal muscular atrophy care in the COVID-19 pandemic era:
Veerapandiyan, A
;
Connolly, A. M
;
Finkel, R. S
...
urn:issn:0148-639X. , 2020
Link:
https://orbi.uliege.be/h..
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7
Effects of Land Cover and Riparian Buffers on Coldwater Fis..:
Sanders, C. L
;
Kinlaw, T
;
Colby, J
...
New River Symposium 2019. , 2019
Link:
http://hdl.handle.net/10..
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8
Nusinersen Versus Sham Control In Infantile-Onset Spinal Mu..:
Finkel, R. S
;
Mercuri, E
;
Darras, B. T
...
10.1056/NEJMoa1702752. , 2017
Link:
http://hdl.handle.net/11..
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9
International consensus guidance for management of myasthen..:
Sanders, D
;
Wolfe, G
;
Benatar, M
...
doi:10.1212/WNL.0000000000002790. , 2016
Link:
https://doi.org/10.1212/..
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10
Pentoxifylline as a rescue treatment for DMD: A randomized ..:
Escolar, D.M
;
Zimmerman, A
;
Bertorini, T
...
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3306159. , 2012
Link:
http://www.ncbi.nlm.nih...
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11
Liquid formulation of pentoxifylline is a poorly tolerated ..:
Zimmerman, A
;
Clemens, P.R
;
Tesi-Rocha, C
...
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3136640. , 2011
Link:
http://www.ncbi.nlm.nih...
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12
Common viruses associated with lower pediatric multiple scl..:
Waubant, E
;
Mowry, E.M
;
Krupp, L
...
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3109881. , 2011
Link:
http://www.ncbi.nlm.nih...
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13
Younger children with MS have a distinct CSF inflammatory p..:
Chabas, D
;
Ness, J
;
Belman, A
...
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2816008. , 2010
Link:
http://www.ncbi.nlm.nih...
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14
P.256 A multicenter, retrospective medical record review of..:
Harvard Medical School, Boston, USA ( host institution )
;
Beggs, A. ( author )
;
Byrne, B. ( UF author )
...
Neuromuscular Disorders. , 2017
Link:
https://doi.org/10.1016/..
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15
P.404 Drug development of vamorolone for Duchenne muscular ..:
University of Pittsburgh, Pittsburgh, USA ( host institution )
;
Clemens, P. ( author )
;
Guglieri, M. ( author )
...
Neuromuscular Disorders. , 2017
Link:
https://doi.org/10.1016/..
1-15