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

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

doi:10.1016/j.nmd.2017.02.013

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

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

E.P Hoffman, Emil Kakkis, Carsten Bonnemann, Petra Kaufmann, Marc Boutin, Jonathan Kimmelman, Bernard Brais, Lawrence Korngut, Filippo Buccella, Joyce Kullman, …

Neuromuscular disorders : NMD, Vol.27(7), pp.693-701

07/2017

DOI: 10.1016/j.nmd.2017.02.013

PMID: 28434909

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Abstract

•Increasing number of drug development programmes present opportunities and challenges.•Increased focus on rigour and de-risking of both pre-clinical and clinical studies.•Focus on lessening burden on patients and families, reducing cost of development.•Increased patient engagement in design, execution, and regulatory processes key.•TREAT-NMD provides an open discussion for these opportunities and challenges.

Details

Title: Subtitle: Facilitating orphan drug development: Proceedings of the TREAT-NMD International Conference, December 2015, Washington, DC, USA
Creators: E.P Hoffman - Binghamton University – SUNY, USA
Emil Kakkis - Novato, CA, USA
Carsten Bonnemann - Bethesda, MD, USA
Petra Kaufmann - Bethesda, MD, USA
Marc Boutin - Washington, DC, USA
Jonathan Kimmelman - Montreal, Quebec, Canada
Bernard Brais - Montreal, Quebec, Canada
Lawrence Korngut - Calgary, Alberta, Canada
Filippo Buccella - Rome, Italy
Joyce Kullman - Kansas City, MO, USA
Hanns Lochmüller - Newcastle, UK
Arthur Burghes - Columbus, OH, USA
Christopher Coffey - Iowa City, IA, USA
Stefano Marini - Rome, Italy
Craig McDonald - Davis, CA, USA
Nabarun Dasgupta - Boston, MA, USA
Hugh Dawkins - Perth, Australia
Charles Mohan - Pittsburgh, PA, USA
Annamaria De Luca - Bari, Italy
Lauren Morgenroth - Washington, DC, USA
Christopher Dowd - Bethesda, MD, USA
Hiroki Morizono - Washington, DC, USA
Tina Duong - Stanford, CA, USA
Kanneboyina Nagaraju - Washington, DC, USA
John Porter - Bethesda, MD, USA
Michelle Eagle - Newcastle, UK
Richard Finkel - Orlando, FL, USA
Lori Reilly - Washington, DC, USA
Pat Furlong - Hackensack, NJ, USA
Markus Rüegg - Basel, Switzerland
Joel Schneider - Boston, MA, USA
Cynthia Gagnon - Montreal, Quebec, Canada
Pietro Spitali - Leiden, The Netherlands
Nathalie Goemans - Leuven, Belgium
Michela Guglieri - Newcastle, UK
Volker Straub - Newcastle, UK
Yetrib Hathout - Washington, DC, USA
Lee Sweeney - Gainesville, FL, USA
Nicholas Johnson - Salt Lake City, UT, USA
Giorgio Tasca - Ottawa, Ontario, Canada
Cathy Turner - Newcastle, UK
Olav Veldhuizen - Newcastle, UK
Jan Verschuuren - Leiden, The Netherlands
Susan Ward - Cambridge, MA, USA
Raffaella Willmann - Basel, Switzerland
Workshop Participants
Resource Type: Journal article
Publication Details: Neuromuscular disorders : NMD, Vol.27(7), pp.693-701
DOI: 10.1016/j.nmd.2017.02.013
PMID: 28434909
NLM abbreviation: Neuromuscul Disord
ISSN: 0960-8966
eISSN: 1873-2364
Publisher: Elsevier B.V
Grant note: DOI: 10.13039/100001625, name: Foundation to Eradicate Duchenne; name: AGADA BioSciences; DOI: 10.13039/100005614, name: Biogen; name: Biomarin; name: Catabasis; DOI: 10.13039/100004312, name: Lilly; name: Idera; DOI: 10.13039/501100014383, name: Italfarmaco; name: MDA USA; DOI: 10.13039/100013223, name: PTC Therapeutics; DOI: 10.13039/100014943, name: Sarepta Therapeutics
Language: English
Date published: 07/2017
Academic Unit: Biostatistics
Record Identifier: 9983997448402771

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