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Journal of Inherited Metabolic Disease
|
May 13, 2024
Clinical pharmacology considerations for first-in-human clinical trials for enzyme replacement therapy
Sydney Stern, Jie Wang, Ruo-Jing Li, et al.
Nature Reviews. Drug Discovery
|
February 2, 2013
Pharmacogenetics in the evaluation of new drugs: a multiregional regulatory perspective
Marc Maliepaard, Charity Nofziger, Marisa Papaluca, et al.
The AAPS Journal
|
January 18, 2023
Model-Informed Approach Supporting Approval of Nexviazyme (Avalglucosidase Alfa-ngpt) in Pediatric Patients with Late-Onset Pompe Disease
Ruo-Jing Li, Lian Ma, Katarzyna Drozda, et al.
Journal of Inherited Metabolic Disease
|
March 17, 2021
Regulatory news: Dojolvi (triheptanoin) as a source of calories and fatty acids in long-chain fatty acid oxidation disorders: FDA approval summary
Dina Zand, Jenny Doan, Sojeong Yi, et al.
Clinical Pharmacology and Therapeutics
|
December 9, 2017
Enrichment Strategies in Pediatric Drug Development: An Analysis of Trials Submitted to the US Food and Drug Administration
Dionna J Green, Xiaomei I Liu, Tianyi Hua, et al.
Hepatology (Baltimore, Md.)
|
April 11, 2012
Response-guided telaprevir therapy in prior relapsers? The role of bridging data from treatment-naïve and experienced subjects
Jiang Liu, Pravin R Jadhav, Shashi Amur, et al.
Hepatology (Baltimore, Md.)
|
May 22, 2012
Boceprevir dosing for late responders and null responders: the role of bridging data between treatment-naïve and -experienced subjects
Jeffry Florian, Pravin R Jadhav, Shashi Amur, et al.
Journal of Clinical Pharmacology
|
December 3, 2022
Model-Informed Approach Supporting Drug Development and Regulatory Evaluation for Rare Diseases
Ruo-Jing Li, Lian Ma, Fang Li, et al.
Journal of Inherited Metabolic Disease
|
August 2, 2021
Regulatory news: Nulibry (fosdenopterin) approved to reduce the risk of mortality in patients with molybdenum cofactor deficiency type A: FDA approval summary
Sheila Farrell, Jacqueline Karp, Rebecca Hager, et al.
Clinical Pharmacology and Therapeutics
|
February 24, 2018
Considerations for Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease
Robert N Schuck, Janet Woodcock, Issam Zineh, et al.
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of 5
Search research articles
Search
Showing results (31-40 of 43) with videos related to
Sort By:
Page
of 5
Journal of Inherited Metabolic Disease
|
May 13, 2024
Clinical pharmacology considerations for first-in-human clinical trials for enzyme replacement therapy
Sydney Stern, Jie Wang, Ruo-Jing Li, et al.
Nature Reviews. Drug Discovery
|
February 2, 2013
Pharmacogenetics in the evaluation of new drugs: a multiregional regulatory perspective
Marc Maliepaard, Charity Nofziger, Marisa Papaluca, et al.
The AAPS Journal
|
January 18, 2023
Model-Informed Approach Supporting Approval of Nexviazyme (Avalglucosidase Alfa-ngpt) in Pediatric Patients with Late-Onset Pompe Disease
Ruo-Jing Li, Lian Ma, Katarzyna Drozda, et al.
Journal of Inherited Metabolic Disease
|
March 17, 2021
Regulatory news: Dojolvi (triheptanoin) as a source of calories and fatty acids in long-chain fatty acid oxidation disorders: FDA approval summary
Dina Zand, Jenny Doan, Sojeong Yi, et al.
Clinical Pharmacology and Therapeutics
|
December 9, 2017
Enrichment Strategies in Pediatric Drug Development: An Analysis of Trials Submitted to the US Food and Drug Administration
Dionna J Green, Xiaomei I Liu, Tianyi Hua, et al.
Hepatology (Baltimore, Md.)
|
April 11, 2012
Response-guided telaprevir therapy in prior relapsers? The role of bridging data from treatment-naïve and experienced subjects
Jiang Liu, Pravin R Jadhav, Shashi Amur, et al.
Hepatology (Baltimore, Md.)
|
May 22, 2012
Boceprevir dosing for late responders and null responders: the role of bridging data between treatment-naïve and -experienced subjects
Jeffry Florian, Pravin R Jadhav, Shashi Amur, et al.
Journal of Clinical Pharmacology
|
December 3, 2022
Model-Informed Approach Supporting Drug Development and Regulatory Evaluation for Rare Diseases
Ruo-Jing Li, Lian Ma, Fang Li, et al.
Journal of Inherited Metabolic Disease
|
August 2, 2021
Regulatory news: Nulibry (fosdenopterin) approved to reduce the risk of mortality in patients with molybdenum cofactor deficiency type A: FDA approval summary
Sheila Farrell, Jacqueline Karp, Rebecca Hager, et al.
Clinical Pharmacology and Therapeutics
|
February 24, 2018
Considerations for Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease
Robert N Schuck, Janet Woodcock, Issam Zineh, et al.
Page
of 5