E-LIB Suche - Ergebnisse für: Swoboda, Kathryn

1

Continued benefit of nusinersen initiated in the presymptom..:

Crawford, Thomas O. ; Swoboda, Kathryn J. ; De Vivo, Darryl C....
Muscle & Nerve. 68 (2023) 2 - p. 157-170 , 2023

Link: https://doi.org/10.1002/..

2

Optimization of base editors for the functional correction ..:

Alves, Christiano R. R. ; Ha, Leillani L. ; Yaworski, Rebecca...
Nature Biomedical Engineering. , 2023

Link: https://doi.org/10.1038/..

3

Baseline Characteristics and Interim Safety in RESPOND: A P..:

Kuntz, Nancy ; Parsons, Julie ; Brandsema, John F....
Neurology. 100 (2023) 17_supplement_2 - p. , 2023

Link: https://doi.org/10.1212/..

4

Population-Based Screening of Newborns: Findings From the N..:

Brower, Amy ; Chan, Kee ; Williams, Marc...
Frontiers in Genetics. 13 (2022) - p. , 2022

Link: https://doi.org/10.3389/..

5

An expanded access program of risdiplam for patients with T..:

Kwon, Jennifer M. ; Arya, Kapil ; Kuntz, Nancy...
Annals of Clinical and Translational Neurology. 9 (2022) 6 - p. 810-818 , 2022

Link: https://doi.org/10.1002/..

6

Baseline Characteristics and Initial Safety Results in RESP..:

Brandsema, John ; Parsons, Julie ; Proud, Crystal...
Neurology. 98 (2022) 18_supplement - p. , 2022

Link: https://doi.org/10.1212/..

7

Onasemnogene abeparvovec for presymptomatic infants with tw..:

Strauss, Kevin A. ; Farrar, Michelle A. ; Muntoni, Francesco...
Nature Medicine. 28 (2022) 7 - p. 1381-1389 , 2022

Link: https://doi.org/10.1038/..

8

Onasemnogene Abeparvovec in Presymptomatic Spinal Muscular ..:

Strauss, Kevin ; Muntoni, Francesco ; Farrar, Michelle...
Neurology. 98 (2022) 18_supplement - p. , 2022

Link: https://doi.org/10.1212/..

9

Muscle microRNAs in the cerebrospinal fluid predict clinica..:

Magen, Iddo ; Aharoni, Sharon ; Yacovzada, Nancy Sarah...
European Journal of Neurology. 29 (2022) 8 - p. 2420-2430 , 2022

Link: https://doi.org/10.1111/..

10

Onasemnogene Abeparvovec for Presymptomatic Infants with Sp..:

Strauss, Kevin ; Muntoni, Francesco ; Farrar, Michelle...
Neurology. 98 (2022) 18_supplement - p. , 2022

Link: https://doi.org/10.1212/..

11

264th ENMC International Workshop: Multi-system involvement..:

Detering, Nora Tula ; Zambon, Alberto ; Hensel, Niko...
Neuromuscular Disorders. 32 (2022) 8 - p. 697-705 , 2022

Link: https://doi.org/10.1016/..

12

Onasemnogene abeparvovec for presymptomatic infants with th..:

Strauss, Kevin A. ; Farrar, Michelle A. ; Muntoni, Francesco...
Nature Medicine. 28 (2022) 7 - p. 1390-1397 , 2022

Link: https://doi.org/10.1038/..

13

Natural history of 10-meter walk/run test performance in sp..:

Krosschell, Kristin J. ; Townsend, Elise L. ; Kiefer, Michael...
Neuromuscular Disorders. 32 (2022) 2 - p. 125-134 , 2022

Link: https://doi.org/10.1016/..

14

Adeno-associated virus serotype 9 antibodies in patients sc..:

Day, John W. ; Finkel, Richard S. ; Mercuri, Eugenio...
Molecular Therapy - Methods & Clinical Development. 21 (2021) - p. 76-82 , 2021

Link: https://doi.org/10.1016/..

15

Implications of circulating neurofilaments for spinal muscu..:

Alves, Christiano R.R. ; Petrillo, Marco ; Spellman, Rebecca...
Molecular Therapy - Methods & Clinical Development. 23 (2021) - p. 524-538 , 2021

Link: https://doi.org/10.1016/..