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.
J. Day, R. Finkel, C. Chiriboga, A. Connolly +17 more
Researcher profile · Semantic Scholar
J. Day, R. Finkel, C. Chiriboga, A. Connolly +17 more
K. Strauss, M. Farrar, F. Muntoni, K. Saito +16 more
K. Strauss, M. Farrar, F. Muntoni, K. Saito +17 more
P. Clemens, V. Rao, A. Connolly, A. Harper +9 more
D. Chand, C. Zaidman, K. Arya, R. Millner +6 more
J. R. Mendell, F. Muntoni, Craig M. McDonald, E. Mercuri +21 more
Ryan Johnson, K. Ng, Alexander R. Dietz, M. Hartman +4 more
E. Mercuri, J. J. Vílchez, O. Boespflug-Tanguy, C. Zaidman +23 more
C. Zaidman, C. Proud, C. McDonald, K. Lehman +14 more
Johan A. Telleman, A. Grimm, S. Goedee, L. Visser +1 more
F. Walker, M. Cartwright, K. Alter, L. Visser +15 more
C. Zaidman, Jim S. Wu, K. Kapur, A. Pasternak +7 more
A. Connolly, Elizabeth C. Malkus, J. Mendell, K. Flanigan +18 more
S. Rutkove, K. Kapur, C. Zaidman, Jim S. Wu +7 more
I. Shklyar, T. Geisbush, Aleksandar S. Mijialovic, A. Pasternak +4 more
S. Rutkove, T. Geisbush, A. Mijailovic, I. Shklyar +5 more
R. Finkel, B. Darras, J. Mendell, John W. Day +13 more
Rocio Garcia-Santibanez, C. Zaidman, R. Sommerville, G. Lopate +3 more
C. Zaidman, Lucy Lu Wang, A. Connolly, J. Florence +8 more
A. Connolly, J. Florence, M. Cradock, M. Eagle +10 more
A. Connolly, C. Zaidman, Paul T. Golumbek, M. Cradock +14 more
I. Fox, Christine B. Novak, Emily M. Krauss, Gwendolyn M. Hoben +5 more
C. Zaidman, Elizabeth C. Malkus, A. Connolly
J. Mendell, Crystal M. Proud, C. Zaidman, Stefanie Mason +7 more
P. Clemens, V. Rao, A. Connolly, A. Harper +6 more
M. Waldrop, S. Moore, K. Mathews, B. Darbro +19 more
C. Zizzi, Elizabeth Luebbe, P. Mongiovi, M. Hunter +9 more
A. Connolly, J. Florence, C. Zaidman, Paul T. Golumbek +20 more
Alexander R. Dietz, Robert C. Bucelli, A. Pestronk, C. Zaidman
R. Potter, I. Moeller, Sohrab Khan, H. Haegel +11 more
K. Ng, Alexander R. Dietz, Ryan Johnson, Michael Shoykhet +1 more
P. Clemens, V. Rao, A. Connolly, A. Harper +6 more
N. Goedeker, Sachi D. Dharia, D. Griffin, Jesantha Coy +6 more
K. Ng, A. Connolly, C. Zaidman
Nirbhay S. Jain, E. Hill, C. Zaidman, C. Novak +7 more
Rocio Garcia-Santibanez, Alexander R. Dietz, Robert C. Bucelli, C. Zaidman
C. Zaidman, N. Goedeker, A. Aqul, R. Butterfield +10 more
A. Dori, H. Abbasi, C. Zaidman
C. Zaidman, Cameron D. Crockett, E. Wedge, G. Tabatabai +1 more
Melanie L. Leitner, K. Kapur, B. Darras, Michele L. Yang +8 more
S. Rutkove, Jim S. Wu, C. Zaidman, K. Kapur +8 more
N. Goedeker, Amanda Rogers, Mark Fisher, K. Arya +17 more
B. Roy, B. Darras, C. Zaidman, Jim S. Wu +2 more
Sisir Koppaka, I. Shklyar, S. Rutkove, Basil T. Darras +3 more
N. Goedeker, J. L. Gibbons, Arun S. Varadhachary, Anne M. Connolly +1 more
R. Govindarajan, S. Iyadurai, A. Connolly, C. Zaidman
A. Connolly, C. Zaidman, John F. Brandsema, Han C. Phan +5 more