E-LIB Suche - Ergebnisse für: Lilien, Charlotte

1

Patients' Perceptions of Nusinersen Effects According to Th..:

Lilien, Charlotte ; Vrscaj, Eva ; Thapaliya, Gita...
Journal of Clinical Medicine. 13 (2024) 12 - p. 3418 , 2024

2

266th ENMC International Workshop: Remote delivery of clini..:

Alfano, Lindsay N. ; James, Meredith K. ; Ramdharry, Gita M....
Neuromuscular Disorders. 33 (2023) 4 - p. 339-348 , 2023

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

3

INCEPTUS Natural History, Run-in Study for Gene Replacement..:

Dowling, James J. ; Müller-Felber, Wolfgang ; Smith, Barbara K....
Journal of Neuromuscular Diseases. 9 (2022) 4 - p. 503-516 , 2022

Link: https://doi.org/10.3233/..

4

Upper limb disease evolution in exon 53 skipping eligible p..:

Lilien, Charlotte ; Reyngoudt, Harmen ; Seferian, Andreea Mihaela...
Annals of Clinical and Translational Neurology. 8 (2021) 10 - p. 1938-1950 , 2021

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

5

Hierarchical Bayesian modelling of disease progression to i..:

the NatHis-MTM Study Group ; Fouarge, Eve ; Monseur, Arnaud...
Orphanet Journal of Rare Diseases. 16 (2021) 1 - p. , 2021

Link: https://doi.org/10.1186/..

6

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/..

7

Natural History of Type 2 and 3 Spinal Muscular Atrophy (SM..:

Servais, Laurent ; Seferian, Andreea Mihaela ; Daron, Aurore...
Neurology. 94 (2020) 15_supplement - p. , 2020

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

8

Natural history of Type 2 and 3 spinal muscular atrophy: 2‐..:

Annoussamy, Mélanie ; Seferian, Andreea M. ; Daron, Aurore...
Annals of Clinical and Translational Neurology. 8 (2020) 2 - p. 359-373 , 2020

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

9

Home-Based Monitor for Gait and Activity Analysis

JoVE : Bioengineering

Lilien, Charlotte ; Gasnier, Erwan ; Gidaro, Teresa... , 2019

Link: https://www.jove.com/t/5..

10

X-linked myotubular myopathy: A prospective international n..:

Annoussamy, Mélanie ; Lilien, Charlotte ; Gidaro, Teresa...
Neurology. 92 (2019) 16 - p. , 2019

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

11

Longitudinal functional and NMR assessment of upper limbs i..:

Hogrel, Jean-Yves ; Wary, Claire ; Moraux, Amélie...
Neurology. 86 (2016) 11 - p. 1022-1030 , 2016

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

12

INCEPTUS Natural History, Run-in Study for Gene Replacement..:

Dowling, James J ; Müller-Felber, Wolfgang ; Smith, Barbara K...
Dowling , J J , Müller-Felber , W , Smith , B K , Bönnemann , C G , Kuntz , N L , Muntoni , F , Servais , L , Alfano , L N , Beggs , A H , Bilder , D A , Blaschek , A , Duong , T , Graham , R J , Jain , M , Lawlor , M W , Lee , J , Coats , J , Lilien , C , Lowes , L P , MacBean , V , Neuhaus , S , Noursalehi , M , Pitts , T , Finlay , C , Christensen , S , Rafferty , G , Seferian , A M , Tsuchiya , E , James , E S , Miller , W , Sepulveda , B , Vila , M C , Prasad , S , Rico , S & Shieh , P B 2022 , ' INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy ' , Journal of Neuromuscular Diseases , vol. 9 , no. 4 , pp. 503-516 . https://doi.org/10.3233/JND-210781. , 2022

Link: https://kclpure.kcl.ac.u..

13

Clinical Trial Readiness for Spinal Muscular Atrophy: Exper..:

Tizzano, Eduardo F ; Christie-Brown, Vanessa ; Baranello, Giovanni...
https://discovery.ucl.ac.uk/id/eprint/10164623/2/Baranello_Tizzano.E%20ScotoM.%20Clinical%20trial%20workshop.revised%20version%201%20-%20VCB.pdf. , 2022

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

14

INCEPTUS Natural History, Run-in Study for Gene Replacement..:

Dowling, James J ; Müller-Felber, Wolfgang ; Smith, Barbara K...
Dowling , J J , Müller-Felber , W , Smith , B K , Bönnemann , C G , Kuntz , N L , Muntoni , F , Servais , L , Alfano , L N , Beggs , A H , Bilder , D A , Blaschek , A , Duong , T , Graham , R J , Jain , M , Lawlor , M W , Lee , J , Coats , J , Lilien , C , Lowes , L P , MacBean , V , Neuhaus , S , Noursalehi , M , Pitts , T , Finlay , C , Christensen , S , Rafferty , G , Seferian , A M , Tsuchiya , E , James , E S , Miller , W , Sepulveda , B , Vila , M C , Prasad , S , Rico , S & Shieh , P B 2022 , ' INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy ' , Journal of Neuromuscular Diseases , vol. 9 , no. 4 , pp. 503-516 . https://doi.org/10.3233/JND-210781. , 2022

Link: https://kclpure.kcl.ac.u..

15

INCEPTUS Natural History, Run-in Study for Gene Replacement..:

Dowling, James J ; Müller-Felber, Wolfgang ; Smith, Barbara K...
https://discovery.ucl.ac.uk/id/eprint/10155738/1/jnd_2022_9-4_jnd-9-4-jnd210781_jnd-9-jnd210781.pdf. , 2022

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