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Author/Creator Finkel, Richard S

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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2. 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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3. Clinical practice guideline for the management of paediatric Charcot-Marie-Tooth disease. Issue 5 (9th February 2022)

Authors:
Yiu, Eppie M; Bray, Paula; Baets, Jonathan; Baker, Steven K; Barisic, Nina; de Valle, Katy; Estilow, Timothy; Farrar, Michelle A; Finkel, Richard S; Haberlová, Jana; Kennedy, Rachel A; Moroni, Isabella; Nicholson, Garth A; Ramchandren, Sindhu; Reilly, Mary M; Rose, Kristy; Shy, Michael E; Siskind...
Journal:
Journal of neurology, neurosurgery and psychiatry
Issue:
Volume 93:Issue 5(2022)
Page Start:
530
Record Type:
Journal Article
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4. Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial. Issue 9979 (2nd May 2015)

Authors:
Buyse, Gunnar M; Voit, Thomas; Schara, Ulrike; Straathof, Chiara S M; D'Angelo, M Grazia; Bernert, Günther; Cuisset, Jean-Marie; Finkel, Richard S; Goemans, Nathalie; McDonald, Craig M; Rummey, Christian; Meier, Thomas
Journal:
Lancet
Issue:
Volume 385:Issue 9979(2015)
Page Start:
1748
Record Type:
Journal Article
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5. 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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6. Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial. Issue 10329 (12th March 2022)

Authors:
McDonald, Craig M; Marbán, Eduardo; Hendrix, Suzanne; Hogan, Nathaniel; Ruckdeschel Smith, Rachel; Eagle, Michelle; Finkel, Richard S; Tian, Cuixia; Janas, Joanne; Harmelink, Matthew M; Varadhachary, Arun S; Taylor, Michael D; Hor, Kan N; Mayer, Oscar H; Henricson, Erik K; Furlong, Pat; Ascheim, ...
Journal:
Lancet
Issue:
Volume 399:Issue 10329(2022)
Page Start:
1049
Record Type:
Journal Article
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7. Selective serotonin reuptake inhibitors ameliorate MEGF10 myopathy. (2nd April 2019)

Authors:
Saha, Madhurima; Rizzo, Skylar A; Ramanathan, Manashwi; Hightower, Rylie M; Santostefano, Katherine E; Terada, Naohiro; Finkel, Richard S; Berg, Jonathan S; Chahin, Nizar; Pacak, Christina A; Wagner, Richard E; Alexander, Matthew S; Draper, Isabelle; Kang, Peter B
Journal:
Human molecular genetics
Issue:
Volume 28:Number 14(2019)
Page Start:
2365
Record Type:
Journal Article
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8. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study. Issue 10063 (17th December 2016)

Authors:
Finkel, Richard S; Chiriboga, Claudia A; Vajsar, Jiri; Day, John W; Montes, Jacqueline; De Vivo, Darryl C; Yamashita, Mason; Rigo, Frank; Hung, Gene; Schneider, Eugene; Norris, Daniel A; Xia, Shuting; Bennett, C Frank; Bishop, Kathie M
Journal:
Lancet
Issue:
Volume 388:Issue 10063(2016)
Page Start:
3017
Record Type:
Journal Article
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9. Treatment of infantile-onset spinal muscular atrophy with nusinersen: final report of a phase 2, open-label, multicentre, dose-escalation study. (July 2021)

Authors:
Finkel, Richard S; Chiriboga, Claudia A; Vajsar, Jiri; Day, John W; Montes, Jacqueline; De Vivo, Darryl C; Bishop, Kathie M; Foster, Richard; Liu, Yingying; Ramirez-Schrempp, Daniela; Schneider, Eugene; Bennett, C Frank; Wong, Janice; Farwell, Wildon
Journal:
Lancet
Issue:
Volume 5:Number 7(2021)
Page Start:
491
Record Type:
Journal Article
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