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Author/Creator Mendell, Jerry R

1. 250 AVXS-101 phase 3 study in spinal muscular atrophy type 1. Issue 12 (14th November 2019)

Authors:
Day, John W; Chiriboga, Claudia A; Crawford, Thomas O; Darras, Basil T; Finkel, Richard S; Connolly, Anne M; Iannaccone, Susan T; Kuntz, Nancy L; Peña, Loren DM; Schultz, Meredith; Shieh, Perry B; Smith, Edward C; Kausar, Imran; Feltner, Douglas E; Ogrinc, Francis G; Ouyang, Haojun; Macek, Thomas...
Journal:
Journal of neurology, neurosurgery and psychiatry
Issue:
Volume 90:Issue 12(2019)
Page Start:
e60
Record Type:
Journal Article
View Content:
Available online (eLD content is only available in our Reading Rooms) ↗

3. Muscle MRI in patients with dysferlinopathy: pattern recognition and implications for clinical trials. Issue 10 (7th May 2018)

Authors:
Diaz-Manera, Jordi; Fernandez-Torron, Roberto; LLauger, Jaume; James, Meredith K; Mayhew, Anna; Smith, Fiona E; Moore, Ursula R; Blamire, Andrew M; Carlier, Pierre G; Rufibach, Laura; Mittal, Plavi; Eagle, Michelle; Jacobs, Marni; Hodgson, Tim; Wallace, Dorothy; Ward, Louise; Smith, Mark; Stramar...
Other Names:
author non-byline.; Arrieta Adrienne author non-byline.; Feng Jia author non-byline.; Hwang Esther author non-byline.; Lee Elaine author non-byline.; Illa Isabel author non-byline.; Gallardo Eduard author non-byline.; Hernández Irene Pedrosa author non-byline.; Jimeno Izaskun Belmonte author non...
Journal:
Journal of neurology, neurosurgery and psychiatry
Issue:
Volume 89:Issue 10(2018)
Page Start:
1071
Record Type:
Journal Article
View Content:
Available online (eLD content is only available in our Reading Rooms) ↗

4. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial. Issue 4 (April 2021)

Authors:
Day, John W; Finkel, Richard S; Chiriboga, Claudia A; Connolly, Anne M; Crawford, Thomas O; Darras, Basil T; Iannaccone, Susan T; Kuntz, Nancy L; Peña, Loren D M; Shieh, Perry B; Smith, Edward C; Kwon, Jennifer M; Zaidman, Craig M; Schultz, Meredith; Feltner, Douglas E; Tauscher-Wisniewski, Sitra...
Journal:
Lancet neurology
Issue:
Volume 20:Issue 4(2021)
Page Start:
284
Record Type:
Journal Article
View Content:
Available online (eLD content is only available in our Reading Rooms) ↗

5. Teenage exercise is associated with earlier symptom onset in dysferlinopathy: a retrospective cohort study. Issue 11 (29th January 2018)

Authors:
Moore, Ursula R; Jacobs, Marni; Fernandez-Torron, Roberto; Jang, Jiji; James, Meredith K; Mayhew, Anna; Rufibach, Laura; Mittal, Plavi; Eagle, Michelle; Cnaan, Avital; Carlier, Pierre G; Blamire, Andrew; Hilsden, Heather; Lochmüller, Hanns; Grieben, Ulrike; Spuler, Simone; Tesi Rocha, Carolina; D...
Journal:
Journal of neurology, neurosurgery and psychiatry
Issue:
Volume 89:Issue 11(2018)
Page Start:
1224
Record Type:
Journal Article
View Content:
Available online (eLD content is only available in our Reading Rooms) ↗