Abstract

P189: Vestronidase alfa for the treatment of mucopolysaccharidosis VII (MPS VII): Updated results from a novel, longitudinal, multicenter disease monitoring program

Full Text
Published version (Free)

Talk to us

Join us for a 30 min session where you can share your feedback and ask us any queries you have

Schedule a call

Similar Papers

Paper Title
Journal
Date
Author
The MPS VII disease monitoring program (DMP) is a novel, longitudinal, cohort program with rigor beyond a traditional registry
Ken Sansone ... Qais Abu Ali
Molecular Genetics and Metabolism | VOL. 126
Ken Sansone, et. al.Ken Sansone ... Qais Abu Ali
01 Feb 2019
Disease characteristics, effectiveness, and safety of vestronidase alfa for the treatment of patients with mucopolysaccharidosis VII in a novel, longitudinal, multicenter disease monitoring program
Roberto Giugliani ... J Lawrence Merritt Ii
Orphanet Journal of Rare Diseases | VOL. 19
Roberto Giugliani, et. al.Roberto Giugliani ... J Lawrence Merritt Ii
07 May 2024
EP166: Vestronidase alfa for the treatment of Mucopolysaccharidosis VII (MPS VII): Updated results from a novel, longitudinal, multicenter disease monitoring program
Deborah Marsden ... Antonio Gonzalez-Meneses Lopez
Genetics in Medicine | VOL. 24
Deborah Marsden, et. al.Deborah Marsden ... Antonio Gonzalez-Meneses Lopez
01 Mar 2022
Vestronidase alfa: Recombinant human β-glucuronidase as an enzyme replacement therapy for MPS VII
Steven Jungles ... Jaclyn Cadaoas
Molecular Genetics and Metabolism | VOL. 130
Steven Jungles, et. al.Steven Jungles ... Jaclyn Cadaoas
06 Mar 2020
View more papers

More From: Genetics in Medicine Open

Paper Title
Journal
Date
Author
Regulatory Elements in SEM1–DLX5–DLX6 (7q21.3) Locus Contribute to Genetic Control of Coronal Nonsyndromic Craniosynostosis and Bone Density-Related Traits
Paola Nicoletti ... Inga Peter
Genetics in Medicine Open | VOL. -
Paola Nicoletti, et. al.Paola Nicoletti ... Inga Peter
01 May 2024
Returning actionable genetic results to participants in the biobank at the Colorado Center for Personalized Medicine and UCHealth
Jan T Lowery ... Kristy R Crooks
Genetics in Medicine Open | VOL. -
Jan T Lowery, et. al.Jan T Lowery ... Kristy R Crooks
01 May 2024
Continuing education and professional development: Unifying opportunities for genetic counselors globally
Kathleen D Valverde ... Daniel J Rader
Genetics in Medicine Open | VOL. -
Kathleen D Valverde, et. al.Kathleen D Valverde ... Daniel J Rader
01 May 2024
Genetic counseling workforce diversity, inclusion, and capacity in Australia and New Zealand
Anaita Kanga-Parabia ... Saraya Hogan
Genetics in Medicine Open | VOL. -
Anaita Kanga-Parabia, et. al.Anaita Kanga-Parabia ... Saraya Hogan
01 May 2024
View more papers