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Clinical and Translational Science
|
December 19, 2025
Surrogate Endpoints in Regulatory Decision-Making
Linda J B Jeng, Jeffrey Siegel
Journal of Inherited Metabolic Disease
|
August 5, 2022
Regulatory news: Avalglucosidase alfa-ngpt (Nexviazyme) for late-onset Pompe disease-FDA approval summary
Ann R Punnoose, Linda J B Jeng, Janet W Maynard, et al.
Drug Safety
|
September 15, 2025
The Development and Use of Office of New Drugs Custom Medical Queries for Safety Analyses of Clinical Trial Data
Scott Proestel, Vaishali Popat, Ellis F Unger, et al.
Clinical Dysmorphology
|
December 5, 2012
2p16.3 microdeletion with partial deletion of the neurexin-1 gene in a female with developmental delays, short stature, and a congenital diaphragmatic hernia
Karla Bermudez-Wagner, Linda J B Jeng, Anne M Slavotinek, et al.
Journal of Inherited Metabolic Disease
|
May 20, 2024
Regulatory news: Cipaglucosidase alfa-atga (Pombiliti) coadministered with Miglustat (Opfolda) for adults with late-onset Pompe disease
Daniela V Luquetti, Linda J B Jeng, Kathleen M Donohue, et al.
Journal of AAPOS : the Official Publication of the American Association for Pediatric Ophthalmology and Strabismus
|
March 27, 2013
Lipodermoid in a patient with Emanuel syndrome
Tanya S Glaser, Katherine A Rauen, Linda J B Jeng, et al.
Therapeutic Innovation & Regulatory Science
|
June 14, 2025
Semantic Search of FDA Guidance Documents Using Generative AI
Scott Proestel, Linda J B Jeng, Christopher Smith, et al.
American Journal of Medical Genetics. Part A
|
February 7, 2014
Novel interstitial 2.6 Mb deletion on 9q21 associated with multiple congenital anomalies
Heather H Pua, Swetha Krishnamurthi, Jessica Farrell, 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.
The Journal of Molecular Diagnostics : JMD
|
April 4, 2024
In Vitro Functional Analysis Can Aid Precision Diagnostics of HNF1B-MODY
Aishwarya Pavithram, Haichen Zhang, Kristin A Maloney, et al.
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Search research articles
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Showing results (1-10 of 16) with videos related to
Sort By:
Page
of 2
Clinical and Translational Science
|
December 19, 2025
Surrogate Endpoints in Regulatory Decision-Making
Linda J B Jeng, Jeffrey Siegel
Journal of Inherited Metabolic Disease
|
August 5, 2022
Regulatory news: Avalglucosidase alfa-ngpt (Nexviazyme) for late-onset Pompe disease-FDA approval summary
Ann R Punnoose, Linda J B Jeng, Janet W Maynard, et al.
Drug Safety
|
September 15, 2025
The Development and Use of Office of New Drugs Custom Medical Queries for Safety Analyses of Clinical Trial Data
Scott Proestel, Vaishali Popat, Ellis F Unger, et al.
Clinical Dysmorphology
|
December 5, 2012
2p16.3 microdeletion with partial deletion of the neurexin-1 gene in a female with developmental delays, short stature, and a congenital diaphragmatic hernia
Karla Bermudez-Wagner, Linda J B Jeng, Anne M Slavotinek, et al.
Journal of Inherited Metabolic Disease
|
May 20, 2024
Regulatory news: Cipaglucosidase alfa-atga (Pombiliti) coadministered with Miglustat (Opfolda) for adults with late-onset Pompe disease
Daniela V Luquetti, Linda J B Jeng, Kathleen M Donohue, et al.
Journal of AAPOS : the Official Publication of the American Association for Pediatric Ophthalmology and Strabismus
|
March 27, 2013
Lipodermoid in a patient with Emanuel syndrome
Tanya S Glaser, Katherine A Rauen, Linda J B Jeng, et al.
Therapeutic Innovation & Regulatory Science
|
June 14, 2025
Semantic Search of FDA Guidance Documents Using Generative AI
Scott Proestel, Linda J B Jeng, Christopher Smith, et al.
American Journal of Medical Genetics. Part A
|
February 7, 2014
Novel interstitial 2.6 Mb deletion on 9q21 associated with multiple congenital anomalies
Heather H Pua, Swetha Krishnamurthi, Jessica Farrell, 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.
The Journal of Molecular Diagnostics : JMD
|
April 4, 2024
In Vitro Functional Analysis Can Aid Precision Diagnostics of HNF1B-MODY
Aishwarya Pavithram, Haichen Zhang, Kristin A Maloney, et al.
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of 2