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(1 - 5 of 5)
Meeting report of the "Regulatory Exchange Matters" session at the 5th International TREAT-NMD Conference: Lessons in communication: How an early dialogue between patients, regulators and academics can further therapy development for neuromuscular disorde
Aartsma-Rus, A.; Mercuri, E.; Vroom, E.; Balabanov, P. 2018
Meeting report of the "Regulatory Exchange Matters" session at the 5th International TREAT-NMD Conference: Lessons in communication: How an early dialogue between patients, regulators and academics can further therapy development for neuromuscular disorders Freiburg, Germany, 27-29 November 2017
Article / Letter to editor
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Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues
Aartsma-Rus, A.; Straub, V.; Hemmings, R.; Haas, M.; Schlosser-Weber, G.; Stoyanova-Beninska, V.; ... ; Balabanov, P. 2017
Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues
Article / Letter to editor
open access
Stakeholder collaboration for spinal muscular atrophy therapy development
Aartsma-Rus, A.; Balabanov, P.; Binetti, L.; Haas, M.; Haberkamp, M.; Mitchell, J.; ... ; Mercuri, E. 2017
Stakeholder collaboration for spinal muscular atrophy therapy development
Article / Letter to editor
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Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy
Straub, V.; Balabanov, P.; Bushby, K.; Ensini, M.; Goemans, N.; Luca, A. de; ... ; Aartsma-Rus, A. 2016
Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy
Article / Letter to editor
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Translational and Regulatory Challenges for Exon Skipping Therapies
Aartsma-Rus, A.; Ferlini, A.; Goemans, N.; Pasmooij, A.M.G.; Wells, D.J.; Bushby, K.; ... ; Balabanov, P. 2014
Translational and Regulatory Challenges for Exon Skipping Therapies
Article / Letter to editor
metadata only