Facilitating orphan drug development: Proceedings of the TREAT-NMD International Conference, December 2015, Washington, DC, USA

Carsten Bonnemann; Marc Boutin; Bernard Brais; Filippo Buccella; Arthur Burghes; Christopher Coffey; Nabarun Dasgupta; Hugh Dawkins; Annamaria De Luca; Christopher Dowd; Tina Duong; Michelle Eagle; Richard Finkel; Pat Furlong; Cynthia Gagnon; Nathalie Goemans; Michela Guglieri; Yetrib Hathout; Nicholas Johnson; Emil Kakkis; Petra Kaufmann; Jonathan Kimmelman; Lawrence Korngut; Joyce Kullman; Hanns Lochmüller; Stefano Marini; Craig McDonald; Charles Mohan; Lauren Morgenroth; Hiroki Morizono; Kanneboyina Nagaraju; John Porter; Lori Reilly; Markus Rüegg; Joel Schneider; Pietro Spitali; Volker Straub; Lee Sweeney; Giorgio Tasca; Cathy Turner; Olav Veldhuizen; Jan Verschuuren; Susan Ward; Raffaella Willmann; Susan Ward

doi:10.1016/j.nmd.2017.02.013

Facilitating orphan drug development: Proceedings of the TREAT-NMD International Conference, December 2015, Washington, DC, USA

Carsten Bonnemann
, Marc Boutin
, Bernard Brais
, Filippo Buccella
, Arthur Burghes
, Christopher Coffey
, Nabarun Dasgupta
, Hugh Dawkins
, Annamaria De Luca
, Christopher Dowd
, Tina Duong
, Michelle Eagle
, Richard Finkel
, Pat Furlong
, Cynthia Gagnon
, Nathalie Goemans
, Michela Guglieri
, Yetrib Hathout
, Nicholas Johnson
, Emil Kakkis

Petra Kaufmann, Jonathan Kimmelman, Lawrence Korngut, Joyce Kullman, Hanns Lochmüller, Stefano Marini, Craig McDonald, Charles Mohan, Lauren Morgenroth, Hiroki Morizono, Kanneboyina Nagaraju, John Porter, Lori Reilly, Markus Rüegg, Joel Schneider, Pietro Spitali, Volker Straub, Lee Sweeney, Giorgio Tasca, Cathy Turner, Olav Veldhuizen, Jan Verschuuren, Susan Ward, Raffaella Willmann, Susan Ward

Research output: Contribution to journal › Article › peer-review

1 Scopus citations

Original language	English
Pages (from-to)	693-701
Number of pages	9
Journal	Neuromuscular Disorders
Volume	27
Issue number	7
DOIs	https://doi.org/10.1016/j.nmd.2017.02.013
State	Published - Jul 2017

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Bonnemann, C., Boutin, M., Brais, B., Buccella, F., Burghes, A., Coffey, C., Dasgupta, N., Dawkins, H., De Luca, A., Dowd, C., Duong, T., Eagle, M., Finkel, R., Furlong, P., Gagnon, C., Goemans, N., Guglieri, M., Hathout, Y., Johnson, N., ... Ward, S. (2017). Facilitating orphan drug development: Proceedings of the TREAT-NMD International Conference, December 2015, Washington, DC, USA. Neuromuscular Disorders, 27(7), 693-701. https://doi.org/10.1016/j.nmd.2017.02.013

@article{8efa515203984e0c8450bc07758f1ffb,

title = "Facilitating orphan drug development: Proceedings of the TREAT-NMD International Conference, December 2015, Washington, DC, USA",

author = "Carsten Bonnemann and Marc Boutin and Bernard Brais and Filippo Buccella and Arthur Burghes and Christopher Coffey and Nabarun Dasgupta and Hugh Dawkins and \{De Luca\}, Annamaria and Christopher Dowd and Tina Duong and Michelle Eagle and Richard Finkel and Pat Furlong and Cynthia Gagnon and Nathalie Goemans and Michela Guglieri and Yetrib Hathout and Nicholas Johnson and Emil Kakkis and Petra Kaufmann and Jonathan Kimmelman and Lawrence Korngut and Joyce Kullman and Hanns Lochm{\"u}ller and Stefano Marini and Craig McDonald and Charles Mohan and Lauren Morgenroth and Hiroki Morizono and Kanneboyina Nagaraju and John Porter and Lori Reilly and Markus R{\"u}egg and Joel Schneider and Pietro Spitali and Volker Straub and Lee Sweeney and Giorgio Tasca and Cathy Turner and Olav Veldhuizen and Jan Verschuuren and Susan Ward and Raffaella Willmann and Susan Ward",

note = "Funding Information: The conference was supported by Foundation to Eradicate Duchenne, AGADA BioSciences, Biogen, Biomarin, Catabasis, Lilly, Idera, Italfarmaco, MDA USA, PTC Therapeutics, and Sarepta Therapeutics.",

year = "2017",

month = jul,

doi = "10.1016/j.nmd.2017.02.013",

language = "English",

volume = "27",

pages = "693--701",

journal = "Neuromuscular Disorders",

issn = "0960-8966",

number = "7",

}

Bonnemann, C, Boutin, M, Brais, B, Buccella, F, Burghes, A, Coffey, C, Dasgupta, N, Dawkins, H, De Luca, A, Dowd, C, Duong, T, Eagle, M, Finkel, R, Furlong, P, Gagnon, C, Goemans, N, Guglieri, M, Hathout, Y, Johnson, N, Kakkis, E, Kaufmann, P, Kimmelman, J, Korngut, L, Kullman, J, Lochmüller, H, Marini, S, McDonald, C, Mohan, C, Morgenroth, L, Morizono, H, Nagaraju, K, Porter, J, Reilly, L, Rüegg, M, Schneider, J, Spitali, P, Straub, V, Sweeney, L, Tasca, G, Turner, C, Veldhuizen, O, Verschuuren, J, Ward, S, Willmann, R & Ward, S 2017, 'Facilitating orphan drug development: Proceedings of the TREAT-NMD International Conference, December 2015, Washington, DC, USA', Neuromuscular Disorders, vol. 27, no. 7, pp. 693-701. https://doi.org/10.1016/j.nmd.2017.02.013

TY - JOUR

T1 - Facilitating orphan drug development

T2 - Proceedings of the TREAT-NMD International Conference, December 2015, Washington, DC, USA

AU - Bonnemann, Carsten

AU - Boutin, Marc

AU - Brais, Bernard

AU - Buccella, Filippo

AU - Burghes, Arthur

AU - Coffey, Christopher

AU - Dasgupta, Nabarun

AU - Dawkins, Hugh

AU - De Luca, Annamaria

AU - Dowd, Christopher

AU - Duong, Tina

AU - Eagle, Michelle

AU - Finkel, Richard

AU - Furlong, Pat

AU - Gagnon, Cynthia

AU - Goemans, Nathalie

AU - Guglieri, Michela

AU - Hathout, Yetrib

AU - Johnson, Nicholas

AU - Kakkis, Emil

AU - Kaufmann, Petra

AU - Kimmelman, Jonathan

AU - Korngut, Lawrence

AU - Kullman, Joyce

AU - Lochmüller, Hanns

AU - Marini, Stefano

AU - McDonald, Craig

AU - Mohan, Charles

AU - Morgenroth, Lauren

AU - Morizono, Hiroki

AU - Nagaraju, Kanneboyina

AU - Porter, John

AU - Reilly, Lori

AU - Rüegg, Markus

AU - Schneider, Joel

AU - Spitali, Pietro

AU - Straub, Volker

AU - Sweeney, Lee

AU - Tasca, Giorgio

AU - Turner, Cathy

AU - Veldhuizen, Olav

AU - Verschuuren, Jan

AU - Ward, Susan

AU - Willmann, Raffaella

AU - Ward, Susan

N1 - Funding Information: The conference was supported by Foundation to Eradicate Duchenne, AGADA BioSciences, Biogen, Biomarin, Catabasis, Lilly, Idera, Italfarmaco, MDA USA, PTC Therapeutics, and Sarepta Therapeutics.

PY - 2017/7

Y1 - 2017/7

UR - https://www.scopus.com/pages/publications/85018567245

U2 - 10.1016/j.nmd.2017.02.013

DO - 10.1016/j.nmd.2017.02.013

M3 - Article

C2 - 28434909

AN - SCOPUS:85018567245

SN - 0960-8966

VL - 27

SP - 693

EP - 701

JO - Neuromuscular Disorders

JF - Neuromuscular Disorders

IS - 7

ER -

Facilitating orphan drug development: Proceedings of the TREAT-NMD International Conference, December 2015, Washington, DC, USA

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