Eteplirsen for the treatment of Duchenne muscular dystrophy
Mendell, Jerry R., Rodino-Klapac, Louise R., Sahenk, Zarife, Roush, Kandice, Bird, Loren, Lowes, Linda P., Alfano, Lindsay, Gomez, Ann Maria, Lewis, Sarah, Kota, Janaiah, Malik, Vinod, Shontz, Kim, Walker, Christopher M., Flanigan, Kevin M., Corridore, Marco, Kean, John R., Allen, Hugh D., Shilling, Chris, Melia, Kathleen R., Sazani, Peter, Saoud, Jay B., Kaye, Edward M.
Published in Annals of neurology (01.11.2013)
Published in Annals of neurology (01.11.2013)
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Journal Article
Evidence-based path to newborn screening for duchenne muscular dystrophy
Mendell, Jerry R., Shilling, Chris, Leslie, Nancy D., Flanigan, Kevin M., al-Dahhak, Roula, Gastier-Foster, Julie, Kneile, Kelley, Dunn, Diane M., Duval, Brett, Aoyagi, Alexander, Hamil, Cindy, Mahmoud, Maha, Roush, Kandice, Bird, Lauren, Rankin, Chelsea, Lilly, Heather, Street, Natalie, Chandrasekar, Ram, Weiss, Robert B.
Published in Annals of neurology (01.03.2012)
Published in Annals of neurology (01.03.2012)
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Journal Article
Effects of Angiotensin-Converting Enzyme Inhibitors and/or Beta Blockers on the Cardiomyopathy in Duchenne Muscular Dystrophy
Viollet, Laurence, PhD, Thrush, Philip T., MD, Flanigan, Kevin M., MD, Mendell, Jerry R., MD, Allen, Hugh D., MD
Published in The American journal of cardiology (01.07.2012)
Published in The American journal of cardiology (01.07.2012)
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Journal Article
Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy
Finkel, Richard S, Flanigan, Kevin M, Wong, Brenda, Bönnemann, Carsten, Sampson, Jacinda, Sweeney, H Lee, Reha, Allen, Northcutt, Valerie J, Elfring, Gary, Barth, Jay, Peltz, Stuart W
Published in PloS one (11.12.2013)
Published in PloS one (11.12.2013)
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Journal Article
The Genotypic and Phenotypic Spectrum of BICD2 Variants in Spinal Muscular Atrophy
Koboldt, Daniel C., Waldrop, Megan A., Wilson, Richard K., Flanigan, Kevin M.
Published in Annals of neurology (01.04.2020)
Published in Annals of neurology (01.04.2020)
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Journal Article
A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy
Mendell, Jerry R, Sahenk, Zarife, Malik, Vinod, Gomez, Ana M, Flanigan, Kevin M, Lowes, Linda P, Alfano, Lindsay N, Berry, Katherine, Meadows, Eric, Lewis, Sarah, Braun, Lyndsey, Shontz, Kim, Rouhana, Maria, Clark, Kelly Reed, Rosales, Xiomara Q, Al-Zaidy, Samiah, Govoni, Alessandra, Rodino-Klapac, Louise R, Hogan, Mark J, Kaspar, Brian K
Published in Molecular therapy (01.01.2015)
Published in Molecular therapy (01.01.2015)
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Journal Article
Genetics and emerging treatments for Duchenne and Becker muscular dystrophy
Wein, Nicolas, Alfano, Lindsay, Flanigan, Kevin M
Published in The Pediatric clinics of North America (01.06.2015)
Published in The Pediatric clinics of North America (01.06.2015)
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Journal Article
Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes
Mendell, Jerry R., Sahenk, Zarife, Al-Zaidy, Samiah, Rodino-Klapac, Louise R., Lowes, Linda P., Alfano, Lindsay N., Berry, Katherine, Miller, Natalie, Yalvac, Mehmet, Dvorchik, Igor, Moore-Clingenpeel, Melissa, Flanigan, Kevin M., Church, Kathleen, Shontz, Kim, Curry, Choumpree, Lewis, Sarah, McColly, Markus, Hogan, Mark J., Kaspar, Brian K.
Published in Molecular therapy (05.04.2017)
Published in Molecular therapy (05.04.2017)
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Journal Article
LTBP4 genotype predicts age of ambulatory loss in duchenne muscular dystrophy
Flanigan, Kevin M., Ceco, Ermelinda, Lamar, Kay-Marie, Kaminoh, Yuuki, Dunn, Diane M., Mendell, Jerry R., King, Wendy M., Pestronk, Alan, Florence, Julaine M., Mathews, Katherine D., Finkel, Richard S., Swoboda, Kathryn J., Gappmaier, Eduard, Howard, Michael T., Day, John W., McDonald, Craig, McNally, Elizabeth M., Weiss, Robert B.
Published in Annals of neurology (01.04.2013)
Published in Annals of neurology (01.04.2013)
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Journal Article
Distinctive patterns of microRNA expression in primary muscular disorders
Eisenberg, Iris, Eran, Alal, Nishino, Ichizo, Moggio, Maurizio, Lamperti, Costanza, Amato, Anthony A, Lidov, Hart G, Kang, Peter B, North, Kathryn N, Mitrani-Rosenbaum, Stella, Flanigan, Kevin M, Neely, Lori A, Whitney, Duncan, Beggs, Alan H, Kohane, Isaac S, Kunkel, Louis M
Published in Proceedings of the National Academy of Sciences - PNAS (23.10.2007)
Published in Proceedings of the National Academy of Sciences - PNAS (23.10.2007)
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Journal Article
Gentamicin-induced readthrough of stop codons in duchenne muscular dystrophy
Malik, Vinod, Rodino-Klapac, Louise R., Viollet, Laurence, Wall, Cheryl, King, Wendy, Al-Dahhak, Roula, Lewis, Sarah, Shilling, Christopher J., Kota, Janaiah, Serrano-Munuera, Carmen, Hayes, John, Mahan, John D., Campbell, Katherine J., Banwell, Brenda, Dasouki, Majed, Watts, Victoria, Sivakumar, Kumaraswamy, Bien-Willner, Ricardo, Flanigan, Kevin M., Sahenk, Zarife, Barohn, Richard J., Walker, Christopher M., Mendell, Jerry R.
Published in Annals of neurology (01.06.2010)
Published in Annals of neurology (01.06.2010)
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Journal Article
Intron mutations and early transcription termination in Duchenne and Becker muscular dystrophy
Waldrop, 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.
Published in Human mutation (01.04.2022)
Published in Human mutation (01.04.2022)
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Journal Article
RNA Interference Inhibits DUX4-induced Muscle Toxicity In Vivo: Implications for a Targeted FSHD Therapy
Wallace, Lindsay M, Liu, Jian, Domire, Jacqueline S, Garwick-Coppens, Sara E, Guckes, Susan M, Mendell, Jerry R, Flanigan, Kevin M, Harper, Scott Q.
Published in Molecular therapy (01.07.2012)
Published in Molecular therapy (01.07.2012)
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Journal Article
Selenoprotein N is required for ryanodine receptor calcium release channel activity in human and zebrafish muscle
Jurynec, Michael J, Xia, Ruohong, Mackrill, John J, Gunther, Derrick, Crawford, Thomas, Flanigan, Kevin M, Abramson, Jonathan J, Howard, Michael T, Grunwald, David Jonah
Published in Proceedings of the National Academy of Sciences - PNAS (26.08.2008)
Published in Proceedings of the National Academy of Sciences - PNAS (26.08.2008)
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Journal Article
Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping
Simmons, Tabatha R., Vetter, Tatyana A., Huang, Nianyuan, Vulin-Chaffiol, Adeline, Wein, Nicolas, Flanigan, Kevin M.
Published in Molecular therapy. Methods & clinical development (11.06.2021)
Published in Molecular therapy. Methods & clinical development (11.06.2021)
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Journal Article
Analysis of Dystrophin Deletion Mutations Predicts Age of Cardiomyopathy Onset in Becker Muscular Dystrophy
Kaspar, Rita Wen, Allen, Hugh D, Ray, Will C, Alvarez, Carlos E, Kissel, John T, Pestronk, Alan, Weiss, Robert B, Flanigan, Kevin M, Mendell, Jerry R, Montanaro, Federica
Published in Circulation. Cardiovascular genetics (01.12.2009)
Published in Circulation. Cardiovascular genetics (01.12.2009)
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Journal Article
Cryptic MHC class I-binding peptides are revealed by aminoglycoside-induced stop codon read-through into the 3′ UTR
Goodenough, Elliot, Robinson, Tara M., Zook, Matthew B., Flanigan, Kevin M., Atkinds, John F., Howard, Michael T., Eisenlohr, Laurence C.
Published in Proceedings of the National Academy of Sciences - PNAS (15.04.2014)
Published in Proceedings of the National Academy of Sciences - PNAS (15.04.2014)
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Journal Article
Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse
Wein, Nicolas, Vetter, Tatyana A., Vulin, Adeline, Simmons, Tabatha R., Frair, Emma C., Bradley, Adrienne J., Gushchina, Liubov V., Almeida, Camila F., Huang, Nianyuan, Lesman, Daniel, Rajakumar, Dhanarajan, Weiss, Robert B., Flanigan, Kevin M.
Published in Molecular therapy. Methods & clinical development (08.09.2022)
Published in Molecular therapy. Methods & clinical development (08.09.2022)
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