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Journal of Pharmaceutical Sciences
|
January 30, 2025
Risk assessment and management strategy of two new NDSRIs in a pharmaceutical drug product for the treatment of a rare disease: From prediction to control
Partha Mukherjee, Xin Yao, Sheela Sitaraman, et al.
Traffic (Copenhagen, Denmark)
|
November 11, 2021
Collagen has a unique SEC24 preference for efficient export from the endoplasmic reticulum
Chung-Ling Lu, Steven Ortmeier, Jon Brudvig, et al.
The Journal of Biological Chemistry
|
May 10, 2021
Endolysosomal N-glycan processing is critical to attain the most active form of the enzyme acid alpha-glucosidase
Nithya Selvan, Nickita Mehta, Suresh Venkateswaran, et al.
Molecular Therapy. Methods & Clinical Development
|
March 5, 2021
Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice
Katherine A White, Hemanth R Nelvagal, Timothy A Poole, et al.
Frontiers in Neurology
|
January 20, 2025
Corrigendum: Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease
Barry J Byrne, Giancarlo Parenti, Benedikt Schoser, et al.
Frontiers in Neurology
|
November 4, 2024
Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease
Barry J Byrne, Giancarlo Parenti, Benedikt Schoser, et al.
Therapeutic Advances in Rare Disease
|
March 2, 2026
Miglustat: a first-in-class enzyme stabilizer for cipaglucosidase alfa for the treatment of late-onset Pompe disease
Robert J Hopkin, Barry J Byrne, Mazen M Dimachkie, et al.
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Search research articles
Search
Showing results (1-10 of 7) with videos related to
Sort By:
Page
of 1
Journal of Pharmaceutical Sciences
|
January 30, 2025
Risk assessment and management strategy of two new NDSRIs in a pharmaceutical drug product for the treatment of a rare disease: From prediction to control
Partha Mukherjee, Xin Yao, Sheela Sitaraman, et al.
Traffic (Copenhagen, Denmark)
|
November 11, 2021
Collagen has a unique SEC24 preference for efficient export from the endoplasmic reticulum
Chung-Ling Lu, Steven Ortmeier, Jon Brudvig, et al.
The Journal of Biological Chemistry
|
May 10, 2021
Endolysosomal N-glycan processing is critical to attain the most active form of the enzyme acid alpha-glucosidase
Nithya Selvan, Nickita Mehta, Suresh Venkateswaran, et al.
Molecular Therapy. Methods & Clinical Development
|
March 5, 2021
Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice
Katherine A White, Hemanth R Nelvagal, Timothy A Poole, et al.
Frontiers in Neurology
|
January 20, 2025
Corrigendum: Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease
Barry J Byrne, Giancarlo Parenti, Benedikt Schoser, et al.
Frontiers in Neurology
|
November 4, 2024
Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease
Barry J Byrne, Giancarlo Parenti, Benedikt Schoser, et al.
Therapeutic Advances in Rare Disease
|
March 2, 2026
Miglustat: a first-in-class enzyme stabilizer for cipaglucosidase alfa for the treatment of late-onset Pompe disease
Robert J Hopkin, Barry J Byrne, Mazen M Dimachkie, et al.
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of 1