检索结果 - Eugene Schneider

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  1. 1
    Nusinersen improves walking distance and reduces fatigue in later‐onset spinal muscular atrophy

    Nusinersen improves walking distance and reduces fatigue in later‐onset spinal muscular atrophy 由 Jacqueline Montes, Sally Dunaway Young, Elena Mazzone, Amy Pasternak, Allan M. Glanzman, Richard S. Finkel, Basil T. Darras, Francesco Muntoni, Eugenio Mercuri, Darryl C. De Vivo, Kathie M. Bishop, Eugene Schneider, C. Frank Bennett, Richard Foster, Wildon Farwell

    出版 2019
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  2. 2
    Antisense Inhibition of Prekallikrein to Control Hereditary Angioedema

    Antisense Inhibition of Prekallikrein to Control Hereditary Angioedema 由 Danny M. Cohn, Nicholas J. Viney, Lauré M. Fijen, Eugene Schneider, Veronica J. Alexander, Shuting Xia, Gwendolyn E. Kaeser, Charvi Nanavati, Brenda F. Baker, Richard S. Geary, Marcel Levi, Joost C.M. Meijers, Erik S.G. Stroes

    出版 2020
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  3. 3
    Clinical effect and safety profile of recombinant human lysosomal acid lipase in patients With cholesteryl ester storage disease

    Clinical effect and safety profile of recombinant human lysosomal acid lipase in patients With cholesteryl ester storage disease 由 Manisha Balwani, Catherine Breen, Gregory M. Enns, Patrick Deegan, Tomáš Honzík, Simon Jones, John P. Kane, Vĕra Malinová, Reena Sharma, Eveline O. Stock, Vassili Valayannopoulos, Ed Wraith, Jennifer M. Burg, Stephen Eckert, Eugene Schneider, Anthony G. Quinn

    出版 2013
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  4. 4
    Nusinersen in later-onset spinal muscular atrophy

    Nusinersen in later-onset spinal muscular atrophy 由 Basil T. Darras, Claudia A. Chiriboga, Susan T. Iannaccone, Kathryn J. Swoboda, Jacqueline Montes, Laurence Mignon, Shuting Xia, C. Frank Bennett, Kathie M. Bishop, Jeremy M. Shefner, Allison Green, Peng Sun, Ishir Bhan, Sarah Gheuens, Eugene Schneider, Wildon Farwell, Darryl C. De Vivo

    出版 2019
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  5. 5
    Inhibition of Prekallikrein for Hereditary Angioedema

    Inhibition of Prekallikrein for Hereditary Angioedema 由 Lauré M. Fijen, Marc A. Riedl, Laura Bordone, Jonathan A. Bernstein, Jason Raasch, Raffi Tachdjian, Timothy Craig, William Lumry, Michael Manning, Veronica J. Alexander, Ken B. Newman, Alexey S. Revenko, Brenda F. Baker, Charvi Nanavati, A. Robert MacLeod, Eugene Schneider, Danny M. Cohn

    出版 2022
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  6. 6
    Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data

    Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data 由 Nicholas J. Viney, Shuling Guo, Bruce L. Tai, Brenda F. Baker, Mariam Aghajan, Shiangtung W. Jung, Rosie Z. Yu, Sheri Booten, Heather Murray, Todd Machemer, Sebastien A. Burel, Sue Murray, Gustavo Büchele, Sotirios Tsimikas, Eugene Schneider, Richard S. Geary, Merrill D. Benson, Brett P. Monia

    出版 2020
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  7. 7
    Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen

    Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen 由 Teresa Coelho, Márcia Waddington‐Cruz, Chi‐Chao Chao, Yeşim Parman, Jonas Wixner, Markus Weiler, Fábio Barroso, Noel R. Dasgupta, Shiangtung W. Jung, Eugene Schneider, Nicholas J. Viney, P. James B. Dyck, Yukio Ando, Julian D. Gillmore, Sami Khella, Morie A. Gertz, Laura Obici, John L. Berk

    出版 2022
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  8. 8
    Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

    Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy 由 Richard S. Finkel, Eugenio Mercuri, Basil T. Darras, Anne M. Connolly, Nancy L. Kuntz, Janbernd Kirschner, Claudia A. Chiriboga, Kayoko Saito, Laurent Servais, Eduardo F. Tizzano, Haluk Topaloğlu, M. Tulinius, Jacqueline Montes, Allan M. Glanzman, Kathie M. Bishop, Zhensbao Zhong, Sarah Gheuens, C. Frank Bennett, Eugene Schneider, Wildon Farwell, Darryl C. De Vivo

    出版 2017
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  9. 9
    Sebelipase alfa over 52weeks reduces serum transaminases, liver volume and improves serum lipids in patients with lysosomal acid lipase deficiency

    Sebelipase alfa over 52weeks reduces serum transaminases, liver volume and improves serum lipids in patients with lysosomal acid lipase deficiency 由 Vassili Valayannopoulos, Vĕra Malinová, Tomáš Honzík, Manisha Balwani, Catherine Breen, Patrick Deegan, Gregory M. Enns, Simon Jones, John P. Kane, Eveline O. Stock, Radhika Tripuraneni, Stephen Eckert, Eugene Schneider, Gavin Hamilton, Michael S. Middleton, Claude B. Sirlin, Bruce Kessler, Christopher Bourdon, Simeon A. Boyadjiev, Reena Sharma, Chris Twelves, Chester B. Whitley, Anthony G. Quinn

    出版 2014
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  10. 10
    Rapid progression and mortality of lysosomal acid lipase deficiency presenting in infants

    Rapid progression and mortality of lysosomal acid lipase deficiency presenting in infants 由 Simon Jones, Vassili Valayannopoulos, Eugene Schneider, Stephen Eckert, Maryam Banikazemi, Martin G. Bialer, Stephen Cederbaum, Alicia Chan, Anil Dhawan, Maja Di Rocco, Jennifer Domm, Gregory M. Enns, David N. Finegold, J. Jay Gargus, Ornella Guardamagna, Christian J. Hendriksz, Iman G. Mahmoud, Julian Raiman, Laila Selim, Chester B. Whitley, Osama K. Zaki, Anthony G. Quinn

    出版 2015
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  11. 11
    Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

    Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy 由 Eugenio Mercuri, Basil T. Darras, Claudia A. Chiriboga, John Day, Craig Campbell, Anne M. Connolly, Susan T. Iannaccone, Janbernd Kirschner, Nancy L. Kuntz, Kayoko Saito, Perry B. Shieh, M. Tulinius, Elena Mazzone, Jacqueline Montes, Kathie M. Bishop, Qingqing Yang, Richard Foster, Sarah Gheuens, C. Frank Bennett, Wildon Farwell, Eugene Schneider, Darryl C. De Vivo, Richard S. Finkel

    出版 2018
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  12. 12
    Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense Oligonucleotide AKCEA-TTR-LRx (ION-682884-CS3) in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

    Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense Oligonucleotide AKCEA-TTR-LRx (ION-682884-CS3) in Hereditary Transthyretin-Mediated Amyloid Polyneuro... 由 Teresa Coelho, Yukio Ando, Merrill D. Benson, John L. Berk, Márcia Waddington‐Cruz, P. James B. Dyck, Julian D. Gillmore, Sami Khella, William J. Litchy, Laura Obici, Cecília Monteiro, Li-Jung Tai, Nicholas J. Viney, Gustavo Büchele, Michela Brambatti, Shiangtung W. Jung, Louis O’Dea, Sotirios Tsimikas, Eugene Schneider, Richard S. Geary, Brett P. Monia, Morie A. Gertz

    出版 2021
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  13. 13
    Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy

    Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy 由 Teresa Coelho, Wilson Marques, Noel R. Dasgupta, Chi‐Chao Chao, Yeşim Parman, Marcondes C. França, Yuh‐Cherng Guo, Jonas Wixner, Long‐Sun Ro, Cristian Calandra, Pedro André Kowacs, John L. Berk, Laura Obici, Fábio Barroso, Markus Weiler, Isabel Conceição, Shiangtung W. Jung, Gustavo Büchele, Michela Brambatti, Jersey Chen, Steven G. Hughes, Eugene Schneider, Nicholas J. Viney, Ahmad Masri, Morie R. Gertz, Yukio Ando, Julian D. Gillmore, Sami Khella, P. James B. Dyck, Márcia Waddington‐Cruz, Anna Mazzeo, Aikaterini Papagianni, Mazen M. Dimachkie, Ioannis Zaganas, Edward Gane, Marco Luigetti, Lucía Galán Dávila, Michelle M. Mezei, Juan González Moreno, Pascal Cintas, Davide Pareyson, Rebecca Traub, Julie Khoury, Conrado J. Estol, Merrilee Needham, David Adams, Michael Polydefkis, Thomas H. Brannagan, Vera Bril, Shahram Attarian, Marcelo Rugiero, B. Jane Distad, Eleni Zamba Papanicolaou, Kon‐Ping Lin, Merrill D. Benson, Morton Scheinberg

    出版 2023
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