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Proceedings of the National Academy of Sciences - PNAS, 2015-06, Vol.112 (23), p.7153-7158
- Hathout, Yetrib
- Brody, Edward
- Clemens, Paula R.
- Cripe, Linda
- DeLisle, Robert Kirk
- Furlong, Pat
- Gordish-Dressman, Heather
- Hache, Lauren
- Henricson, Erik
- Hoffman, Eric P.
- Kobayashi, Yvonne Monique
- Lorts, Angela
- Mah, Jean K.
- McDonald, Craig
- Mehler, Bob
- Nelson, Sally
- Nikrad, Malti
- Singer, Britta
- Steele, Fintan
- Sterling, David
- Sweeney, H. Lee
- Williams, Steve
- Gold, Larry
2015
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Health and quality of life outcomes, 2021-07, Vol.19 (1), p.1-184, Article 184
2021
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Frontiers in medicine, 2019-05, Vol.6, p.110-110
2019
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Muscle & nerve, 2020-01, Vol.61 (1), p.36-43
2020
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Expert opinion on orphan drugs, 2014-11, Vol.2 (11), p.1221-1223
2014
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Nature biotechnology, 2019-04, Vol.37 (4), p.352-355
2019
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Health and quality of life outcomes, 2021-07, Vol.19 (1)
2021
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Molecular therapy, 2021-04, Vol.29 (4), p.1367-1369
2021
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European journal of human genetics : EJHG, 2016-05, Vol.24 (5), p.633-637
2016
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The Lancet (British edition), 2022-03, Vol.399 (10329), p.1049-1058
- 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, Deborah D
- Rogy, Siegfried
- Williams, Paula
- Marbán, Linda
- Butterfield, Russell
- Connolly, Anne
- Muntoni, Francesco
- Joyce, Nanette C.
- Evans, Maya
- Abedi, Mehrdad
- Surampudi, Prasanth
- Jhawar, Sanjay
- Dayan, Jonathan G.
- Anthonisen, Colleen
- Goude, Erica
- Nicorici, Alina
- Sarwary, Omaid
- Prasad, Poonam
- Baek, Jayoon
- Newton, Andrew
- Johnson, Hannah
- Kusmik, Kyle
- Filar, Lauri
- Edmondson, Angie
- Rybalsky, Irina
- Chouteau, Wendy
- Giordano, Anthony F.
- Rodriguez, Aixa
- Anderson, Kristan
- Wezel, Germaine
- Vega, Melisa
- Duke, Julie
- Collado, Jorge
- Civitello, Matthew
- Wells, Julie
- Pyzik, Erika
- Rehborg, Rebecca
- Brown, Michelle
- Van Eyk, Jennifer
- Rogers, Russell G.
2022
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Lancet neurology, 2016-07, Vol.15 (8), p.882-890
- Straub, Volker, MD
- Balabanov, Pavel, PhD
- Bushby, Kate, MD
- Ensini, Monica, PhD
- Goemans, Nathalie, MD
- De Luca, Annamaria, PhD
- Pereda, Alejandra
- Hemmings, Robert
- Campion, Giles, MD
- Kaye, Edward, MD
- Arechavala-Gomeza, Virginia, PhD
- Goyenvalle, Aurelie, PhD
- Niks, Erik, MD
- Veldhuizen, Olav
- Furlong, Pat
- Stoyanova-Beninska, Violeta, MD
- Wood, Matthew J, PhD
- Johnson, Alex
- Mercuri, Eugenio, MD
- Muntoni, Francesco, MD
- Sepodes, Bruno, PhD
- Haas, Manuel, PharmD
- Vroom, Elizabeth, DMD
- Aartsma-Rus, Annemieke, Dr
2016
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Neuromuscular disorders : NMD, 2020-10, Vol.30, p.S168-S169
2020
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Neuromuscular disorders : NMD, 2020-10, Vol.30, p.S168-S169
2020
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Neuromuscular disorders : NMD, 2018-08, Vol.28 (8), p.690-701
- Straub, Volker
- Mercuri, Eugenio
- Aartsma-Rus, Annemieke
- Athanasiou, Dimitrios
- Balabanov, Pavel
- Buccella, Filippo
- Caizergues, Didier
- Carlier, Pierre
- Crossley, Emily
- Connolly, Anne M.
- Duong, Tina
- Fischer, Dirk
- Furlong, Pat
- Goemans, Nathalie
- de Groot, Imelda
- Guglieri, Michela
- Henricson, Erik
- Johnson, Alex
- Kan, Hermien E.
- Mayhew, Anna
- Mazzone, Elena
- McDonald, Craig M.
- Muntoni, Francesco
- Niks, Erik H.
- Servais, Laurent
- Turner, Cathy
- Voit, Thomas
- Vroom, Elizabeth
- Walter, Glenn
2018
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The patient : patient-centered outcomes research, 2021-09, Vol.14 (5), p.687-690
2021
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Neuromuscular disorders : NMD, 2015-01, Vol.25 (1), p.96-105
2015
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Orphanet journal of rare diseases, 2015-02, Vol.10 (1), p.16-16
2015
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Journal of neuromuscular diseases, 2024-01, Vol.11 (2), p.499-523
2024
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Neuromuscular disorders : NMD, 2019-08, Vol.29 (8), p.634-643
- Bourke, John P.
- Guglieri, Michela
- Duboc, Denis
- Aartsma-Rus, Annemieke
- Bandali, Alykhan
- Bennett, Neil
- Cools, Bjorn
- Cripe, Linda
- de Groot, Imelda
- Dittrich, Sven
- Florian, Anca
- Furlong, Pat
- Goemans, Nathalie
- Hor, Kan
- van Leperen, Frank
- MacGowan, Guy
- McNally, Elizabeth
- Pegoraro, Elena
- Politano, Luisa
- Sediva, Marie
- Stara, Veronika
- Timmermans, Janneke
- Vroom, Elizabeth
- Wahbi, Karim
2019
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Neuromuscular disorders : NMD, 2016-06, Vol.26 (6), p.386-393
- Bergsma, Arjen
- Lobo-Prat, Joan
- Vroom, Elizabeth
- Furlong, Pat
- Herder, Just L.
- Corrigan, Madeline
- de Groot, Imelda
- Faisal, Aldo
- Goemans, Nathalie
- Han, Jay
- Herder, Just
- Iodice, Mario
- Kennedy, Annie
- Koopman, Bart
- Lobo Prat, Joan
- Main, Marion
- Mathie, Blake
- Muntoni, Francesco
- Castro, Miguel Nobre
- Paalman, Micha
- Porter, John
- Rahman, Tariq
- Schneider, Joel
- Stienen, Arno
- Verstegen, Paul
- Walsh, Conor
2016
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Journal of neuromuscular diseases, 2024-01, Vol.11 (1), p.233-252
2024
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Clinical and translational science, 2021-01, Vol.14 (1), p.94-101
- Izmailova, Elena S.
- Wagner, John A.
- Ammour, Nadir
- Amondikar, Ninad
- Bell‐Vlasov, Andrea
- Berman, Steven
- Bloomfield, Dan
- Brady, Linda S.
- Cai, Xuemei
- Calle, Roberto A.
- Campbell, Michelle
- Cerreta, Francesca
- Clay, Ieuan
- Foschini, Luca
- Furlong, Pat
- Goldel, Rob
- Goldsack, Jennifer S.
- Groenen, Peter M.A.
- Folarin, Amos
- Heemskerk, Jill
- Honig, Peter
- Hotopf, Matthew
- Kamphaus, Tania
- Karlin, Daniel R.
- Leptak, Christopher
- Liu, Qi
- Manji, Husseini
- Mather, Robert J.
- Menetski, Joseph P.
- Narayan, Vaibhav A.
- Papadopoulos, Elektra
- Patel, Bakul
- Patrick‐Lake, Bray
- Podichetty, Jagdeep T.
- Pratap, Abhishek
- Servais, Laurent
- Stephenson, Diane
- Tenaerts, Pam
- Tromberg, Bruce J.
- Usdin, Steve
- Vasudevan, Srikanth
- Zipunnikov, Vadim
- Hoffmann, Steven C.
2021
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Neuromuscular disorders : NMD, 2019-10, Vol.29 (10), p.800-810
2019
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Applied clinical trials, 2016-02, Vol.25 (2/3), p.20
2016
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Zeitschrift / Serie
- Neuromuscular Disorders : Nmd
- Applied Clinical Trials
- Health And Quality Of Life Outcomes
- Journal Of Neuromuscular Diseases
- Plos Currents
- The Journal Of Molecular Diagnostics : Jmd
- Clinical And Translational Science
- Controlled Clinical Trials
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- European Journal Of Human Genetics : Ejhg