E-LIB Suche - Ergebnisse für: GOEMANS, N

1

Efficacy and Safety of Vamorolone vs Placebo and Prednisone..:

Guglieri, M ; Clemens, PR ; Perlman, SJ...
issn:2168-6149. , 2022

Link: http://hdl.handle.net/11..

2

Human iPSC model reveals a central role for NOX4 and oxidat..:

Duelen R ; Costamagna D ; Gilbert G...
info:eu-repo/semantics/altIdentifier/pmid/35090586. , 2022

Link: http://hdl.handle.net/11..

3

Efficacy and Safety of Vamorolone vs Placebo and Prednisone..:

Guglieri, M ; Clemens, PR ; Perlman, SJ...
https://discovery.ucl.ac.uk/id/eprint/10155962/1/Baranello_Efficacy%20and%20Safety%20of%20Vamorolone%20vs%20Placebo%20and%20Prednisone%20among%20Boys%20with%20Duchenne%20Muscular%20Dystrophy_VoR.pdf. , 2022

Link: https://discovery.ucl.ac..

4

Ataluren delays loss of ambulation and respiratory decline ..:

McDonald, CM ; Muntoni, F ; Penematsa, V...
https://discovery.ucl.ac.uk/id/eprint/10139096/1/cer-2021-0196.pdf. , 2021

Link: https://discovery.ucl.ac..

5

North Star Ambulatory Assessment changes in ambulant Duchen..:

Coratti G ; Pane M ; Brogna C...
info:eu-repo/semantics/altIdentifier/pmid/34170974. , 2021

Link: http://hdl.handle.net/24..

6

North Star Ambulatory Assessment changes in ambulant Duchen..:

Coratti G ; Pane M ; Brogna C...
info:eu-repo/semantics/altIdentifier/pmid/34170974. , 2021

Link: http://hdl.handle.net/11..

7

North Star Ambulatory Assessment changes in ambulant Duchen..:

Coratti, G ; Pane, M ; Brogna, C...
https://discovery.ucl.ac.uk/id/eprint/10130664/1/journal.pone.0253882.pdf. , 2021

Link: https://discovery.ucl.ac..

8

Meta-analyses of ataluren randomized controlled trials in n..:

Campbell, C ; Barohn, RJ ; Bertini, E...
issn:2042-6305. , 2020

Link: http://hdl.handle.net/11..

9

Meta-analyses of ataluren randomized controlled trials in n..:

Campbell, C ; Barohn, RJ ; Bertini, E...
https://discovery.ucl.ac.uk/id/eprint/10109632/1/cer-2020-0095.pdf. , 2020

Link: https://discovery.ucl.ac..

10

European ad-hoc consensus statement on gene replacement the..:

Kirschner, J ; Butoianu, N ; Goemans, N...
https://discovery.ucl.ac.uk/id/eprint/10110722/1/European%20ad-hoc%20consensus%20statement%20on%20gene%20replacement%20therapy%20for%20spinal%20muscular%20atrophy.pdf. , 2020

Link: https://discovery.ucl.ac..

11

Age and baseline values predict 12 and 24-month functional ..:

Coratti, G ; Pera, MC ; Lucibello, S...
https://discovery.ucl.ac.uk/id/eprint/10112464/3/Muntoni_Age%20and%20baseline%20values%20predict%2012%20and%2024-month%20functional%20changes%20in%20type%202%20SMA_AAM.pdf. , 2020

Link: https://discovery.ucl.ac..

12

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..

13

Gain and loss of abilities in type II SMA: A 12-month natur..:

Coratti, G ; Lucibello, S ; Pera, MC...
https://discovery.ucl.ac.uk/id/eprint/10110917/1/Muntoni_manuscript%20may%2015.pdf. , 2020

Link: https://discovery.ucl.ac..

14

Learning about learning: An exploration into the effects of..:

Goemans, N
https://ora.ox.ac.uk/objects/uuid:cc82b5df-cc3e-49e9-b87f-88df27c2eb49. , 2020

Link: https://ora.ox.ac.uk/obj..

15

A phase 3 randomized placebo-controlled trial of tadalafil ..:

Victor, RG ; Sweeney, HL ; Finkel, R...
doi:10.1212/wnl.0000000000004570. , 2020

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