The impact on children and parents of participation in clinical research trials for Morquio syndrome type A and Sanfilippo syndrome type A

2014 ◽  
Vol 111 (2) ◽  
pp. S32-S33
Author(s):  
Tara Clancy ◽  
Maria Farag ◽  
Deborah Holliday ◽  
Catherine Breen ◽  
Simon Jones
Keyword(s):  
Clinical Research ◽  
Type A ◽  
Syndrome Type ◽  
Sanfilippo Syndrome ◽  
Children And Parents ◽  
Morquio Syndrome ◽  
Clinical Research Trials ◽  
The Impact

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

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

Cardiac Ultrasound Findings in Sanfilippo Syndrome Type A

2012 ◽  
Vol 105 (2) ◽  
pp. S34
Author(s):  
Brandon Hays ◽  
Elizabeth Braunlin ◽  
James Berry ◽  
Brenda Diethelm-Okita ◽  
Chester Whitley
Keyword(s):  
Type A ◽  
Cardiac Ultrasound ◽  
Syndrome Type ◽  
Sanfilippo Syndrome ◽  
Ultrasound Findings

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

Genotype, Disease Onset, And Neurocognitive Phenotype Of Sanfilippo Syndrome Type A

2012 ◽  
Vol 105 (2) ◽  
pp. S58 ◽  
Author(s):  
Chester B. Whitley ◽  
Renee Cooksley ◽  
Brenda Diethelm-Okita ◽  
Kathleen Delaney ◽  
Patrick Haslett ◽  
...  
Keyword(s):  
Disease Onset ◽  
Type A ◽  
Syndrome Type ◽  
Sanfilippo Syndrome
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