“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

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

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

Multi-stakeholder engagement leading to access to treatment for MPS IVA (Morquio syndrome type A), a model for the ultra rare disease community

2017 ◽  
Vol 120 (1-2) ◽  
pp. S115 ◽  
Author(s):  
Charlotte Roberts ◽  
Christine Lavery ◽  
Nigel Nicholls ◽  
Mohit Jain ◽  
Christian J Hendriksz ◽  
...  
Keyword(s):  
Rare Disease ◽  
Stakeholder Engagement ◽  
Type A ◽  
Syndrome Type ◽  
Access To Treatment ◽  
Mps Iva ◽  
Morquio Syndrome ◽  
Multi Stakeholder ◽  
Disease Community

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

Analysis of C6S/C4S ratio in Morquio syndrome type A patients

2015 ◽  
Vol 114 (2) ◽  
pp. S66
Author(s):  
Francyne Kubaski ◽  
Tsutomu Shimada ◽  
Robert W. Mason ◽  
Sherlly L. Xie ◽  
Ana C. Brusius-Facchin ◽  
...  
Keyword(s):  
Type A ◽  
Syndrome Type ◽  
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

The Morquio A Registry Study (MARS): Improving the understanding of Morquio syndrome type A

2015 ◽  
Vol 114 (2) ◽  
pp. S49 ◽  
Author(s):  
Sue Graham ◽  
Abigail Waite ◽  
Omowunmi Loba-Olopade ◽  
JoEllen Hunt ◽  
Adrian Quartel
Keyword(s):  
Type A ◽  
Syndrome Type ◽  
Registry Study ◽  
Morquio A ◽  
Morquio Syndrome
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