Author/Creator: Day, John W - British Library Electronic Legal Deposit Catalogue-Journal Issues and Articles Search Results

1. 188 SUNFISH 3-year efficacy and safety of risdiplam in types 2 and 3 SMA. Issue 9 (12th August 2022)

Authors:: Servais, Laurent; Day, John W; Mazzone, Elena S; Nascimento, Andres; Oskoui, Maryam; Baranello, Giovanni; Gerber, Marianne; Martin, Carmen; Yeung, Wai Yin; Mercuri, Eugenio
Journal:: Journal of neurology, neurosurgery and psychiatry
Issue:: Volume 93:Issue 9(2022)
Page Start:: e2
Record Type:: Journal Article
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2. 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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3. 255 SUNFISH part 2: risdiplam in type 2 and type 3 SMA. Issue 6 (27th May 2022)

Authors:: Servais, Laurent; Mercuri, Eugenio; Barisic, Nina; Boespflug-Tanguy, Odile; Deconinck, Nicolas; Fuerst-Recktenwald, Sabine; Fuhrer, Sibylle; Gerber, Marianne; Gorni, Ksenija; Day, John W
Journal:: Journal of neurology, neurosurgery and psychiatry
Issue:: Volume 93:Issue 6(2022)
Page Start:: A87
Record Type:: Journal Article
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4. Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial. Issue 3 (March 2023)

Authors:: Thornton, Charles A; Moxley, Richard Thomas; Eichinger, Katy; Heatwole, Chad; Mignon, Laurence; Arnold, W David; Ashizawa, Tetsuo; Day, John W; Dent, Gersham; Tanner, Matthew K; Duong, Tina; Greene, Ericka P; Herbelin, Laura; Johnson, Nicholas E; King, Wendy; Kissel, John T; Leung, Doris G; Lott,...
Journal:: Lancet neurology
Issue:: Volume 22:Issue 3(2023)
Page Start:: 218
Record Type:: Journal Article
View Content:: Available online (eLD content is only available in our Reading Rooms) ↗

6. 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) ↗

7. 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) ↗

8. 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) ↗

9. Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial. Issue 1 (January 2022)

Authors:: Mercuri, Eugenio; Deconinck, Nicolas; Mazzone, Elena S; Nascimento, Andres; Oskoui, Maryam; Saito, Kayoko; Vuillerot, Carole; Baranello, Giovanni; Boespflug-Tanguy, Odile; Goemans, Nathalie; Kirschner, Janbernd; Kostera-Pruszczyk, Anna; Servais, Laurent; Gerber, Marianne; Gorni, Ksenija; Khwaja, ...
Journal:: Lancet neurology
Issue:: Volume 21:Issue 1(2022)
Page Start:: 42
Record Type:: Journal Article
View Content:: Available online (eLD content is only available in our Reading Rooms) ↗

10. Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial. Issue 1 (January 2022)

Authors:: Mercuri, Eugenio; Deconinck, Nicolas; Mazzone, Elena S; Nascimento, Andres; Oskoui, Maryam; Saito, Kayoko; Vuillerot, Carole; Baranello, Giovanni; Boespflug-Tanguy, Odile; Goemans, Nathalie; Kirschner, Janbernd; Kostera-Pruszczyk, Anna; Servais, Laurent; Gerber, Marianne; Gorni, Ksenija; Khwaja, ...
Journal:: Lancet neurology
Issue:: Volume 21:Issue 1(2022)
Page Start:: 42
Record Type:: Journal Article
View Content:: Available online (eLD content is only available in our Reading Rooms) ↗

1. 188 SUNFISH 3-year efficacy and safety of risdiplam in types 2 and 3 SMA. Issue 9 (12th August 2022)

2. 250 AVXS-101 phase 3 study in spinal muscular atrophy type 1. Issue 12 (14th November 2019)

3. 255 SUNFISH part 2: risdiplam in type 2 and type 3 SMA. Issue 6 (27th May 2022)

4. Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial. Issue 3 (March 2023)

5. Mississippi Delta Restoration : Pathways to a sustainable future /: Pathways to a sustainable future. (2017)

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

7. 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)

8. 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)

9. Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial. Issue 1 (January 2022)

10. Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial. Issue 1 (January 2022)

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