The patient experience of elosulfase alfa treatment for MPS IVA (Morquio syndrome type A): beyond traditional measures

2017 ◽  
Vol 120 (1-2) ◽  
pp. S70-S71
Author(s):  
James Jarrett ◽  
Mohit Jain ◽  
Charlotte Roberts ◽  
Alexandra Morrison
Keyword(s):  
Patient Experience ◽  
Type A ◽  
Syndrome Type ◽  
Mps Iva ◽  
Morquio Syndrome ◽  
Elosulfase Alfa

Impact of two-year elosulfase alfa treatment on patient-reported outcomes in patients with Morquio syndrome type A: Results from an English managed access agreement

2019 ◽  
Vol 126 (2) ◽  
pp. S19
Author(s):  
Jacqueline Adam ◽  
Maureen Cleary ◽  
Christian Hendriksz ◽  
Tarekegn Geberhiwot ◽  
Derralynn Hughes ◽  
...  
Keyword(s):  
Patient Reported Outcomes ◽  
Type A ◽  
Syndrome Type ◽  
Patient Reported ◽  
Morquio Syndrome ◽  
Elosulfase Alfa

Airway stenting in MPS IVA (Morquio syndrome type A)

2019 ◽  
Vol 126 (2) ◽  
pp. S156
Author(s):  
Mildrid Yeo ◽  
Sergei Korenev ◽  
Richard Hewitt ◽  
Derek Roebuck ◽  
Maureen Cleary ◽  
...  
Keyword(s):  
Type A ◽  
Syndrome Type ◽  
Airway Stenting ◽  
Mps Iva ◽  
Morquio Syndrome

Elosulfase alfa treatment and changes in physical functioning and disability in Morquio syndrome type A

2017 ◽  
Vol 120 (1-2) ◽  
pp. S65 ◽  
Author(s):  
Christian J. Hendriksz ◽  
Rossella Parini ◽  
Moeenaldeen D. AlSayed ◽  
Julian Raiman ◽  
Roberto Giugliani ◽  
...  
Keyword(s):  
Physical Functioning ◽  
Type A ◽  
Syndrome Type ◽  
Morquio Syndrome ◽  
Elosulfase Alfa

Elosulfase alfa decreases glycosaminoglycan storage in white blood cells from Morquio syndrome type A patients undergoing enzyme replacement

2015 ◽  
Vol 114 (2) ◽  
pp. S17-S18
Author(s):  
Guilherme Baldo ◽  
Fabiano Poswar ◽  
Andressa Federhen ◽  
Rejane Gus ◽  
Fernanda Bender ◽  
...  
Keyword(s):  
Blood Cells ◽  
White Blood Cells ◽  
Type A ◽  
Syndrome Type ◽  
Enzyme Replacement ◽  
Morquio Syndrome ◽  
Elosulfase Alfa

Impact of elosulfase alfa in patients with Morquio syndrome type A who have limited ambulation: An open-label, phase 2 study

2016 ◽  
Vol 117 (2) ◽  
pp. S55
Author(s):  
Paul R. Harmatz ◽  
Andrea Jester ◽  
Eugen Mengel ◽  
Marsha Treadwell ◽  
Barbara K. Burton ◽  
...  
Keyword(s):  
Type A ◽  
Syndrome Type ◽  
Phase 2 ◽  
Open Label ◽  
Phase 2 Study ◽  
Open Label Phase ◽  
Morquio Syndrome ◽  
Elosulfase Alfa

Impact of long-term elosulfase alfa treatment on pulmonary function in patients with Morquio syndrome type A

2016 ◽  
Vol 117 (2) ◽  
pp. S56 ◽  
Author(s):  
Christian J. Hendriksz ◽  
Barbara K. Burton ◽  
Moeen D. AlSayed ◽  
Roberto Giugliani ◽  
Norberto Guelbert ◽  
...  
Keyword(s):  
Pulmonary Function ◽  
Type A ◽  
Syndrome Type ◽  
Morquio Syndrome ◽  
Elosulfase Alfa

Clinical outcomes in Morquio syndrome type A treated with elosulfase alfa: results from the managed access agreement in England

2018 ◽  
Vol 123 (2) ◽  
pp. S67
Author(s):  
Derralynn Hughes ◽  
Maureen Cleary ◽  
Tarekegn Geberhiwot ◽  
Christian J. Hendriksz ◽  
Simon A. Jones ◽  
...  
Keyword(s):  
Clinical Outcomes ◽  
Type A ◽  
Syndrome Type ◽  
Morquio Syndrome ◽  
Elosulfase Alfa

“Little people, big world”: Morquio syndrome type A (MPS IVA) in adult life

2020 ◽  
Vol 129 (2) ◽  
pp. S137
Author(s):  
Tatiane C. Roncato ◽  
Bruno P. Coghi ◽  
Bruna I.B. Silva ◽  
Bruna S. Pastre ◽  
Laura Vagnini ◽  
...  
Keyword(s):  
Adult Life ◽  
Type A ◽  
Syndrome Type ◽  
Mps Iva ◽  
Morquio Syndrome

Long-term clinical outcomes of patients with Morquio syndrome type A treated with elosulfase alfa: Results from a managed access agreement in England

2020 ◽  
Vol 129 (2) ◽  
pp. S114
Author(s):  
Swati Mukherjee ◽  
James Davison ◽  
Rachel Gould ◽  
Tarekegn Geberhiwot ◽  
Derralynn Hughes ◽  
...  
Keyword(s):  
Clinical Outcomes ◽  
Type A ◽  
Syndrome Type ◽  
Morquio Syndrome ◽  
Elosulfase Alfa
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