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Kostera-Pruszczyk, Anna
249  results:
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1

2024 update: European consensus statement on gene therapy f..:

Kirschner, Janbernd ; Bernert, Günther ; Butoianu, Nina...
European Journal of Paediatric Neurology.  51 (2024)  - p. 73-78 , 2024
Link: https://doi.org/10.1016/..
 
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2

Correction to: Analysis of muscle magnetic resonance imagin..:

Esteller, Diana ; Schiava, Marianela ; Verdú-Díaz, José...
Journal of Neurology.  271 (2024)  4 - p. 2147-2148 , 2024
Link: https://doi.org/10.1007/..
 
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3

JEWELFISH: 24-month results from an open-label study in non..:

Chiriboga, Claudia A. ; Bruno, Claudio ; Duong, Tina...
Journal of Neurology.  , 2024
Link: https://doi.org/10.1007/..
 
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4

Analysis of muscle magnetic resonance imaging of a large co..:

Esteller, Diana ; Schiava, Marianela ; Verdú-Díaz, José...
Journal of Neurology.  270 (2023)  12 - p. 5849-5865 , 2023
Link: https://doi.org/10.1007/..
 
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5

Two-year efficacy and safety of risdiplam in patients with ..:

Oskoui, Maryam ; Day, John W. ; Deconinck, Nicolas...
Journal of Neurology.  270 (2023)  5 - p. 2531-2546 , 2023
Link: https://doi.org/10.1007/..
 
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6

Safety and Efficacy of Ataluren in nmDMD Patients from Stud..:

McDonald, Craig M. ; Wu, Shiwen ; Gulati, Sheffali...
Neurology.  100 (2023)  17_supplement_2 - p. , 2023
Link: https://doi.org/10.1212/..
 
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7

Risdiplam in Patients Previously Treated with Other Therapi..:

Chiriboga, Claudia A. ; Bruno, Claudio ; Duong, Tina...
Neurology and Therapy.  12 (2023)  2 - p. 543-557 , 2023
Link: https://doi.org/10.1007/..
 
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8

Correction to: Risdiplam in Patients Previously Treated wit..:

Chiriboga, Claudia A. ; Bruno, Claudio ; Duong, Tina...
Neurology and Therapy.  12 (2023)  5 - p. 1799-1801 , 2023
Link: https://doi.org/10.1007/..
 
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9

Sunfish parts 1 and 2: 4-year efficacy and safety data of r..:

Oskoui, Maryam ; Deconinck, Nicolas ; Mazzone, Elena S....
Journal of the Neurological Sciences.  455 (2023)  - p. 121096 , 2023
Link: https://doi.org/10.1016/..
 
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10

Dysregulated iron homeostasis in dystrophin-deficient cardi..:

Andrysiak, Kalina ; Machaj, Gabriela ; Priesmann, Dominik...
Cardiovascular Research.  120 (2023)  1 - p. 69-81 , 2023
Link: https://doi.org/10.1093/..
 
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11

Efficacy and safety of avalglucosidase alfa in participants..:

Kishnani, Priya S. ; Díaz-Manera, Jordi ; Kushlaf, Hani...
Molecular Genetics and Metabolism.  138 (2023)  2 - p. 107185 , 2023
Link: https://doi.org/10.1016/..
 
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12

Long-term nusinersen treatment across a wide spectrum of sp..:

Łusakowska, Anna ; Wójcik, Adrianna ; Frączek, Anna...
Orphanet Journal of Rare Diseases.  18 (2023)  1 - p. , 2023
Link: https://doi.org/10.1186/..
 
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13

Correction to: Two‑year efficacy and safety of risdiplam in..:

Oskoui, Maryam ; Day, John W. ; Deconinck, Nicolas...
Journal of Neurology.  270 (2023)  5 - p. 2547-2549 , 2023
Link: https://doi.org/10.1007/..
 
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14

Anoctamin-5 related muscle disease: clinical and genetic fi..:

de Bruyn, Alexander ; Montagnese, Federica ; Holm-Yildiz, Sonja...
Brain.  146 (2023)  9 - p. 3800-3815 , 2023
Link: https://doi.org/10.1093/..
 
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15

SUNFISH Parts 1 and 2: 4-year Efficacy and Safety Data of R..:

Servais, Laurent ; Oskoui, Maryam ; Day, John...
Neurology.  100 (2023)  17_supplement_2 - p. , 2023
Link: https://doi.org/10.1212/..
 
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