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Thomas O. Crawford

Johns Hopkins Hospital

78H-index
278Paper Count
2.2WCitation Count
Published Papers 96
Publication Date
New SMA era: A broad-range tiered assessment of function for the evolving SMA phenotype (EVOLVE-SMA)
err2025-12-30
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PREAI
errMeghan Moore Burk; Thomas O Crawford; Kristen Johnson; Susan Apkon; Laurey Brown; Terri Carry; Katie Hoffman; Carolyn Kelley; Nancy L Kuntz; Julie Miller; Jessica Nance; Ruchee Patel; Vamshi Rao; Carly Rothman; Abigail Schwaede; Anne Stratton; Sara Thinger; Jessica Trenkle; Madeline Van Os; Kerry Vela; Tina Duong; Kristin J Krosschell
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A plain language summary of the SAPPHIRE clinical trial of apitegromab in children and young adults with spinal muscular atrophy
err2025-11-13
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errOAAI
errThomas O. Crawford; Laurent Servais; Eugenio Mercuri; Heike Kölbel; Nancy Kuntz; Richard S. Finkel; Jena Krueger; Kaitlin Batley; Sally Dunaway Young; Jing L. Marantz; Guochen Song; Bert Yao; Guolin Zhao; Jose Rossello; Giridhar S. Tirucherai; Elena Stacy Mazzone; Russell J. Butterfield; Marta Gomez Garcia de la Banda; Andreea M. Seferian; Valeria A. Sansone; Liesbeth De Waele; W. Ludo van der Pol; Claude Cances; Astrid Pechmann; Basil T. Darras
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A prospective, multi-center, observational study of the safety, tolerability and effectiveness of nusinersen in adult patients with Spinal Muscular Atrophy
err2025-10-24
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PREAI
errCraig M. Zaidman; Crystal Proud; Bing M. Liao; Nassim Rad; Doreen Ho; Mary-Lynn Chu; Shafeeq Ladha; Thomas O. Crawford; Shakti Nayar; Angela Genge; Margaret Frey; Chad Heatwole; Daphne Lew
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Consensus recommendations and considerations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophy
err2025-08-23
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PREAI
errJodi M. Wolff; Nora Capocci; Evrim Atas; Diana X. Bharucha-Goebel; John F. Brandsema; Russell J. Butterfield; Christina B. Chadwick; Manuela Corti; Thomas O. Crawford; Linda Cripe; John W. Day; Tina Duong; Mai K. ElMallah; Kevin M. Flanigan; Lindsey A. George; Natalie L. Goedeker; Erica Goude; Sharon Hesterlee; Brian Lin; Natalie K. Katz; Susan E. Matesanz; Craig McDonald; Elizabeth M. McNally; Claudia Mercado-Rodriguez; Deipanjan Nandi; Julie A. Parsons; Crystal Proud; Leigh Ramos-Platt; Angela Lek; Stephanie M. Salabarria; Eric Camino; Rachel Schrader; Erin Shea; Richard Shell; Perry B. Shieh; Jonathan H. Soslow; Jane B. Taylor; Aravindhan Veerapandiyan; Chet Villa; Michele L. Yang; Craig M. Zaidman; Carmen Leon-Astudillo; Barry J. Byrne
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Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial
err2025-08-13
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PREAI
errThomas O Crawford; Laurent Servais; Eugenio Mercuri; Heike Kölbel; Nancy Kuntz; Richard S Finkel; Jena Krueger; Kaitlin Batley; Sally Dunaway Young; Jing L Marantz; Guochen Song; Bert Yao; Guolin Zhao; Jose Rossello; Giridhar S Tirucherai; Elena Stacy Mazzone; Russell J Butterfield; Marta Gomez Garcia de la Banda; Andreea M Seferian; Valeria A Sansone; Liesbeth De Waele; W Ludo van der Pol; Claude Cances; Astrid Pechmann; Basil T Darras
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Patient-reported assessment of bulbar function in spinal muscular atrophy (SMA): Validation of a self-report scale
err2025-03-20
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PREAI
errLefton-Greif, Maureen A.; Belter, Lisa; Jarecki, Jill; Schroth, Mary; Zeng, Yong; Crawford, Thomas O.; Eaton, Cyd
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A reassessment of spinal cord pathology in severe infantile spinal muscular atrophy
err2024-10-24
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errOAAI
errAllardyce, Hazel; Lawrence, Benjamin D.; Crawford, Thomas O.; Sumner, Charlotte J.; Parson, Simon H.
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Combined clinical, structural and cellular studies discriminate pathogenic and benign TRPV4 variants
errBRAIN
IF11.7
err2024-07-18
err1
PREAI
errBerth, Sarah H.; Vo, Linh; Kwon, Do Hoon; Grider, Tiffany; Damayanti, Yasmine S.; Kosmanopoulos, Gage; Fox, Andrew; Lau, Alexander R.; Carr, Patrice; Donohue, Jack K.; Hoke, Maya; Thomas, Simone; Karam, Chafic; Fay, Alex J.; Meltzer, Ethan; Crawford, Thomas O.; Gaudet, Rachelle; Shy, Michael E.; Hellmich, Ute A.; Lee, Seok-Yong; Sumner, Charlotte J.; Mccray, Brett A.
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TRPV4 neuromuscular disease registry highlights bulbar, skeletal and proximal limb manifestations
errBRAIN
IF11.7
err2024-06-25
err1
PREAI
errKosmanopoulos, Gage P.; Donohue, Jack K.; Hoke, Maya; Thomas, Simone; Peyton, Margo A.; Vo, Linh; Crawford, Thomas O.; Sadjadi, Reza; Herrmann, David N.; Yum, Sabrina W.; Reilly, Mary M.; Scherer, Steven S.; Finkel, Richard S.; Lewis, Richard A.; Pareyson, Davide; Pisciotta, Chiara; Walk, David; Shy, Michael E.; Sumner, Charlotte J.; Mccray, Brett A.
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Intrathecal Gene Therapy for Giant Axonal Neuropathy
err2024-03-21
err9
PREAI
errBharucha-Goebel, Diana X.; Todd, Joshua J.; Saade, Dimah; Norato, Gina; Jain, Minal; Lehky, Tanya; Bailey, Rachel M.; Chichester, Jessica A.; Calcedo, Roberto; Armao, Diane; Foley, A. Reghan; Mohassel, Payam; Tesfaye, Eshetu; Carlin, Bradley P.; Seremula, Beth; Waite, Melissa; Zein, Wadih M.; Huryn, Laryssa A.; Crawford, Thomas O.; Sumner, Charlotte J.; Hoke, Ahmet; Heiss, John D.; Charnas, Lawrence; Hooper, Jody E.; Bouldin, Thomas W.; Kang, Elizabeth M.; Rybin, Denis; Gray, Steven J.; Bonnemann, Carsten G.
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Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3
err2024-03-12
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errOAAI
errCrawford, Thomas O.; Darras, Basil T.; Day, John W.; Young, Sally Dunaway; Duong, Tina; Nelson, Leslie L.; Barrett, Doreen; Song, Guochen; Bilic, Sanela; Cote, Shaun; Sadanowicz, Mara; Iarrobino, Ryan; Xu, Tiina J.; O'Neil, Janet; Rossello, Jose; Place, Amy; Kertesz, Nathalie; Nomikos, George; Chyung, Yung
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Identifying Biomarkers of Spinal Muscular Atrophy for Further Development
err2023-09-08
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errOAAI
errGlascock, Jacqueline; Darras, Basil T.; Crawford, Thomas O.; Sumner, Charlotte J.; Kolb, Stephen J.; DiDonato, Christine; Elsheikh, Bakri; Howell, Kelly; Farwell, Wildon; Valente, Marta; Petrillo, Marco; Tingey, Jessica; Jarecki, Jill
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Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)
err2023-05-02
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errOAAI
errFinkel, Richard S.; Darras, Basil T.; Mendell, Jerry R.; Day, John W.; Kuntz, Nancy L.; Connolly, Anne M.; Zaidman, Craig M.; Crawford, Thomas O.; Butterfield, Russell J.; Shieh, Perry B.; Tennekoon, Gihan; Brandsema, John F.; Iannaccone, Susan T.; Shoffner, John; Kavanagh, Sarah; Macek, Thomas A.; Tauscher-Wisniewski, Sitra
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Spinraza in Adults with Spinal Muscular Atrophy (SAS)-14 month results
err2023-04-25
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PREAI
errZaidman, Craig; Proud, Crystal; Thonhoff, Jason; Rad, Nassim; Ho, Doreen; Chu, MaryLynn; Ladha, Shafeeq; Crawford, Thomas; Nayar, Shakti; Genge, Angela; Frey, Margaret; Heatwole, Chad; Lew, Daphne
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Mapping gas around massive galaxies: cross-correlation of DES Y3 galaxies and Compton-y maps from SPT and Planck
err2023-04-20
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errOAAI
errSanchez, J.; Omori, Y.; Chang, C.; Bleem, L. E.; Crawford, T.; Drlica-Wagner, A.; Raghunathan, S.; Zacharegkas, G.; Abbott, T. M. C.; Aguena, M.; Alarcon, A.; Allam, S.; Alves, O.; Amon, A.; Avila, S.; Baxter, E.; Bechtol, K.; Benson, B. A.; Bernstein, G. M.; Bertin, E.; Bocquet, S.; Brooks, D.; Burke, D. L.; Campos, A.; Carlstrom, J. E.; Rosell, A. Carnero; Kind, M. Carrasco; Carretero, J.; Castander, F. J.; Cawthon, R.; Chang, C. L.; Chen, A.; Choi, A.; Chown, R.; Costanzi, M.; Crites, A. T.; Crocce, M.; da Costa, L. N.; Pereira, M. E. S.; de Haan, T.; De Vicente, J.; DeRose, J.; Desai, S.; Diehl, H. T.; Dobbs, M. A.; Dodelson, S.; Doel, P.; Elvin-Poole, J.; Everett, W.; Everett, S.; Ferrero, I; Flaugher, B.; Fosalba, P.; Frieman, J.; Garcia-Bellido, J.; Gatti, M.; George, E. M.; Gerdes, D. W.; Giannini, G.; Gruen, D.; Gruendl, R. A.; Gschwend, J.; Gutierrez, G.; Halverson, N. W.; Hinton, S. R.; Holder, G. P.; Hollowood, D. L.; Holzapfel, W. L.; Honscheid, K.; Hrubes, J. D.; James, D. J.; Knox, L.; Kuehn, K.; Kuropatkin, N.; Lahav, O.; Lee, A. T.; Luong-Van, D.; MacCrann, N.; Marshall, J. L.; McCullough, J.; McMahon, J. J.; Melchior, P.; Mena-Fernandez, J.; Menanteau, F.; Miquel, R.; Mocanu, L.; Mohr, J. J.; Muir, J.; Myles, J.; Natoli, T.; Padin, S.; Palmese, A.; Pandey, S.; Paz-Chinchon, F.; Pieres, A.; Malagon, A. A. Plazas; Porredon, A.; Pryke, C.; Raveri, M.; Reichardt, C. L.; Rodriguez-Monroy, M.; Ross, A. J.; Ruhl, J. E.; Rykoff, E.; Sanchez, C.; Sanchez, E.; Scarpine, V; Schaffer, K. K.; Sevilla-Noarbe, I; Sheldon, E.; Shirokoff, E.; Smith, M.; Soares-Santos, M.; Staniszewski, Z.; Stark, A. A.; Suchyta, E.; Swanson, M. E. C.; Tarle, G.; Thomas, D.; Troxel, M. A.; Tucker, D. L.; Vieira, J. D.; Vincenzi, M.; Weaverdyck, N.; Williamson, R.; Yanny, B.; Yin, B.
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Early treatment is a lifeline for infants with SMA
err2022-07-15
err8
errOAAI
errSumner, Charlotte J.; Crawford, Thomas O.
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Intron mutations and early transcription termination in Duchenne and Becker muscular dystrophy
err2022-03-07
err27
errOAAI
errWaldrop, Megan A.; Moore, Steven A.; Mathews, Katherine D.; Darbro, Benjamin W.; Medne, Livja; Finkel, Richard; Connolly, Anne M.; Crawford, Thomas O.; Drachman, Daniel; Wein, Nicolas; Habib, Ali A.; Krzesniak-Swinarska, Monika A.; Zaidman, Craig M.; Collins, James J.; Jokela, Manu; Udd, Bjarne; Day, John W.; Ortiz-Guerrero, Gloria; Statland, Jeff; Butterfield, Russell J.; Dunn, Diane M.; Weiss, Robert B.; Flanigan, Kevin M.
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TRPV4 mutations causing mixed neuropathy and skeletal phenotypes result in severe gain of function
err2022-02-16
err17
errOAAI
errTaga, Arens; Peyton, Margo A.; Goretzki, Benedikt; Gallagher, Thomas Q.; Ritter, Ann; Harper, Amy; Crawford, Thomas O.; Hellmich, Ute A.; Sumner, Charlotte J.; McCray, Brett A.
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Relationship of pharmacokinetics and pharmacodynamics to apitegromab efficacy in patients with later-onset spinal muscular atrophy (Types 2 and 3 SMA): Results from the TOPAZ study
err2021-10-01
err0
PREAI
errCrawford, Thomas; Place, Amy; Barrett, Doreen; Cote, Shaun; Nomikos, George; Song, Guochen; Bilic, Sanela; Kalra, Ashish; Sadanowicz, Mara; O'Neil, Janet; Iarrobino, Ryan; Kertesz, Nathalie; Chyung, Yung
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Clinical and genetic spectrum of a large cohort of patients with δ-sarcoglycan muscular dystrophy
errBRAIN
IF11.7
err2021-09-13
err16
errOAAI
errAlonso-Perez, Jorge; Gonzalez-Quereda, Lidia; Bruno, Claudio; Panicucci, Chiara; Alavi, Afagh; Nafissi, Shahriar; Nilipour, Yalda; Zanoteli, Edmar; de Augusto Isihi, Lucas Michielon; Melegh, Bela; Hadzsiev, Kinga; Muelas, Nuria; Vilchez, Juan J.; Dourado, Mario Emilio; Kadem, Naz; Kutluk, Gultekin; Umair, Muhammad; Younus, Muhammad; Pegorano, Elena; Bello, Luca; Crawford, Thomas O.; Suarez-Calvet, Xavier; Topf, Ana; Guglieri, Michela; Marini-Bettolo, Chiara; Gallano, Pia; Straub, Volker; Diaz-Manera, Jordi
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