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Journal of Women'S Health (2002)
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October 20, 2017
Representation of Women and Minorities in Clinical Trials for New Molecular Entities and Original Therapeutic Biologics Approved by FDA CDER from 2013 to 2015
Alice Chen, Hilary Wright, Hawi Itana, et al.
Pediatrics
|
June 23, 2010
Therapies for inborn errors of metabolism: what has the orphan drug act delivered?
Sonali S Talele, Kui Xu, Anne R Pariser, et al.
Drug Discovery Today
|
November 9, 2025
Non-oncologic orphan drug approvals across the world: Types of evidence required and time to approval
Anne R Pariser, Violeta Stoyanova-Beninska, Oxana Iliach, et al.
Nature Reviews. Drug Discovery
|
November 1, 2011
Clinical pharmacology as a cornerstone of orphan drug development
Edward D Bashaw, Shiew-Mei Huang, Timothy R Coté, et al.
Orphanet Journal of Rare Diseases
|
October 22, 2021
The IDeaS initiative: pilot study to assess the impact of rare diseases on patients and healthcare systems
Ainslie Tisdale, Christine M Cutillo, Ramaa Nathan, et al.
Orphanet Journal of Rare Diseases
|
May 10, 2023
Towards the international interoperability of clinical research networks for rare diseases: recommendations from the IRDiRC Task Force
Rima Nabbout, Galliano Zanello, Dixie Baker, et al.
Orphanet Journal of Rare Diseases
|
September 11, 2024
Operational description of rare diseases: a reference to improve the recognition and visibility of rare diseases
Chiuhui Mary Wang, Amy Heagle Whiting, Ana Rath, et al.
Human Gene Therapy
|
February 20, 2025
Adeno-Associated Virus Gene Therapy Development: Early Planning and Regulatory Considerations to Advance the Platform Vector Gene Therapy Program
Richa Madan Lomash, Jean Dehdashti, Oleg A Shchelochkov, et al.
Molecular Genetics and Metabolism
|
November 25, 2015
Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction
Priya S Kishnani, Patricia I Dickson, Laurie Muldowney, et al.
Systems Medicine (New Rochelle, N.Y.)
|
April 1, 2020
Recent Advances in Systems and Network Medicine: Meeting Report from the First International Conference in Systems and Network Medicine
Emma Kurnat-Thoma, Ancha Baranova, Pat Baird, et al.
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of 3
Search research articles
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Showing results (11-20 of 21) with videos related to
Sort By:
Page
of 3
Journal of Women'S Health (2002)
|
October 20, 2017
Representation of Women and Minorities in Clinical Trials for New Molecular Entities and Original Therapeutic Biologics Approved by FDA CDER from 2013 to 2015
Alice Chen, Hilary Wright, Hawi Itana, et al.
Pediatrics
|
June 23, 2010
Therapies for inborn errors of metabolism: what has the orphan drug act delivered?
Sonali S Talele, Kui Xu, Anne R Pariser, et al.
Drug Discovery Today
|
November 9, 2025
Non-oncologic orphan drug approvals across the world: Types of evidence required and time to approval
Anne R Pariser, Violeta Stoyanova-Beninska, Oxana Iliach, et al.
Nature Reviews. Drug Discovery
|
November 1, 2011
Clinical pharmacology as a cornerstone of orphan drug development
Edward D Bashaw, Shiew-Mei Huang, Timothy R Coté, et al.
Orphanet Journal of Rare Diseases
|
October 22, 2021
The IDeaS initiative: pilot study to assess the impact of rare diseases on patients and healthcare systems
Ainslie Tisdale, Christine M Cutillo, Ramaa Nathan, et al.
Orphanet Journal of Rare Diseases
|
May 10, 2023
Towards the international interoperability of clinical research networks for rare diseases: recommendations from the IRDiRC Task Force
Rima Nabbout, Galliano Zanello, Dixie Baker, et al.
Orphanet Journal of Rare Diseases
|
September 11, 2024
Operational description of rare diseases: a reference to improve the recognition and visibility of rare diseases
Chiuhui Mary Wang, Amy Heagle Whiting, Ana Rath, et al.
Human Gene Therapy
|
February 20, 2025
Adeno-Associated Virus Gene Therapy Development: Early Planning and Regulatory Considerations to Advance the Platform Vector Gene Therapy Program
Richa Madan Lomash, Jean Dehdashti, Oleg A Shchelochkov, et al.
Molecular Genetics and Metabolism
|
November 25, 2015
Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction
Priya S Kishnani, Patricia I Dickson, Laurie Muldowney, et al.
Systems Medicine (New Rochelle, N.Y.)
|
April 1, 2020
Recent Advances in Systems and Network Medicine: Meeting Report from the First International Conference in Systems and Network Medicine
Emma Kurnat-Thoma, Ancha Baranova, Pat Baird, et al.
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of 3