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Author/Creator Shieh, Perry B

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
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Available online (eLD content is only available in our Reading Rooms) ↗

2. Amifampridine phosphate (Firdapse®) is effective and safe in a phase 3 clinical trial in LEMS. Issue 5 (3rd March 2016)

Authors:
Oh, Shin J; Shcherbakova, Natalya; Kostera‐Pruszczyk, Anna; Alsharabati, Mohammad; Dimachkie, Mazen; Blanco, Jose Munoz; Brannagan, Thomas; Lavrnić, Dragana; Shieh, Perry B; Vial, Christophe; Meisel, Andreas; Komoly, Samuel; Schoser, Benedikt; Sivakumar, Kumaraswamy; So, Yuen
Journal:
Muscle & nerve
Issue:
Volume 53:Issue 5(2016)
Page Start:
717
Record Type:
Journal Article
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Available online (eLD content is only available in our Reading Rooms) ↗

3. Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Issue 10101 (23rd September 2017)

Authors:
McDonald, Craig M; Campbell, Craig; Torricelli, Ricardo Erazo; Finkel, Richard S; Flanigan, Kevin M; Goemans, Nathalie; Heydemann, Peter; Kaminska, Anna; Kirschner, Janbernd; Muntoni, Francesco; Osorio, Andrés Nascimento; Schara, Ulrike; Sejersen, Thomas; Shieh, Perry B; Sweeney, H Lee; Topaloglu...
Journal:
Lancet
Issue:
Volume 390:Issue 10101(2017)
Page Start:
1489
Record Type:
Journal Article
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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
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Available online (eLD content is only available in our Reading Rooms) ↗

5. Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial. Issue 12 (December 2021)

Authors:
Diaz-Manera, Jordi; Kishnani, Priya S; Kushlaf, Hani; Ladha, Shafeeq; Mozaffar, Tahseen; Straub, Volker; Toscano, Antonio; van der Ploeg, Ans T; Berger, Kenneth I; Clemens, Paula R; Chien, Yin-Hsiu; Day, John W; Illarioshkin, Sergey; Roberts, Mark; Attarian, Shahram; Borges, Joao Lindolfo; Bouhou...
Journal:
Lancet neurology
Issue:
Volume 20:Issue 12(2021)
Page Start:
1012
Record Type:
Journal Article
View Content:
Available online (eLD content is only available in our Reading Rooms) ↗