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Pat Furlong

Showing results (11-20 of 26) with videos related to

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Nature Biotechnology|April 4, 2019
What compassionate use means for gene therapiesCarolyn Riley Chapman, Kenneth I Moch, Andrew McFadyen, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|March 14, 2021
Gene therapy companies have an ethical obligation to develop expanded access policiesLisa Kearns, Carolyn Riley Chapman, Kenneth I Moch, et al.
Orphanet Journal of Rare Diseases|March 12, 2015
Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpointsEmil D Kakkis, Mary O'Donovan, Gerald Cox, et al.
Journal of Neuromuscular Diseases|February 16, 2024
Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Related Dystrophinopathies - Developing Potential Treatments for the Entire Spectrum of DiseaseCraig McDonald, Eric Camino, Rafael Escandon, et al.
Orphanet Journal of Rare Diseases|June 25, 2015
How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug AdministrationPat Furlong, John F P Bridges, Lawrence Charnas, et al.
Journal of Multidisciplinary Healthcare|September 3, 2024
Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy TherapiesRyan Fischer, Pat Furlong, Annie Kennedy, et al.
Neurology|October 4, 2017
A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophyRonald G Victor, H Lee Sweeney, Richard Finkel, et al.
Neuromuscular Disorders : NMD|October 14, 2014
Measuring clinical effectiveness of medicinal products for the treatment of Duchenne muscular dystrophyStephen Lynn, Annemieke Aartsma-Rus, Kate Bushby, et al.
The Journal of Molecular Diagnostics : JMD|March 1, 2011
Quality assurance for Duchenne and Becker muscular dystrophy genetic testing: development of a genomic DNA reference material panelLisa Kalman, Jay Leonard, Norman Gerry, et al.
Journal of Neuromuscular Diseases|February 20, 2025
A Parent Project Muscular Dystrophy-sponsored International Workshop Report on Endocrine and Bone Issues in Patients with Duchenne Muscular Dystrophy: An Ever-changing LandscapeLeanne M Ward, David R Weber, Sze Choong Wong, et al.
Pageof 3

Showing results (11-20 of 26) with videos related to

Sort By:
Pageof 3
Nature Biotechnology|April 4, 2019
What compassionate use means for gene therapiesCarolyn Riley Chapman, Kenneth I Moch, Andrew McFadyen, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|March 14, 2021
Gene therapy companies have an ethical obligation to develop expanded access policiesLisa Kearns, Carolyn Riley Chapman, Kenneth I Moch, et al.
Orphanet Journal of Rare Diseases|March 12, 2015
Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpointsEmil D Kakkis, Mary O'Donovan, Gerald Cox, et al.
Journal of Neuromuscular Diseases|February 16, 2024
Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Related Dystrophinopathies - Developing Potential Treatments for the Entire Spectrum of DiseaseCraig McDonald, Eric Camino, Rafael Escandon, et al.
Orphanet Journal of Rare Diseases|June 25, 2015
How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug AdministrationPat Furlong, John F P Bridges, Lawrence Charnas, et al.
Journal of Multidisciplinary Healthcare|September 3, 2024
Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy TherapiesRyan Fischer, Pat Furlong, Annie Kennedy, et al.
Neurology|October 4, 2017
A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophyRonald G Victor, H Lee Sweeney, Richard Finkel, et al.
Neuromuscular Disorders : NMD|October 14, 2014
Measuring clinical effectiveness of medicinal products for the treatment of Duchenne muscular dystrophyStephen Lynn, Annemieke Aartsma-Rus, Kate Bushby, et al.
The Journal of Molecular Diagnostics : JMD|March 1, 2011
Quality assurance for Duchenne and Becker muscular dystrophy genetic testing: development of a genomic DNA reference material panelLisa Kalman, Jay Leonard, Norman Gerry, et al.
Journal of Neuromuscular Diseases|February 20, 2025
A Parent Project Muscular Dystrophy-sponsored International Workshop Report on Endocrine and Bone Issues in Patients with Duchenne Muscular Dystrophy: An Ever-changing LandscapeLeanne M Ward, David R Weber, Sze Choong Wong, et al.
Pageof 3