Author Search Results

1

Clinical relevance of endpoints in clinical trials for acid sphingomyelinase deficiency enzyme replacement therapy

by Jones, Simon A.

Published 2020

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2

Obstructive Sleep Apnea in MPS: A Systematic Review of Pretreatment and Posttreatment Prevalence and Severity

by Pal,Abhijit Ricky

Published 2015

Other Authors: “...Jones,Simon A....”
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3

Long-Term Galsulfase Treatment Associated With Improved Survival of Patients With Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome): 15-Year Follow-Up From the Survey Study

by Quartel,Adrian

Published 2018

Other Authors: “...Jones,Simon A....”
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4

Toward a Core Outcome Set for Head, Neck, and Respiratory Disease in Mucopolysaccharidosis Type II: Systematic Literature Review and Assessment of Heterogeneity in Outcome Reportin...

by Metryka,Aleksandra

Published 2019

Other Authors: “...Jones,Simon...”
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5

Long-term galsufase treatment associated with improved survival of patients with mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome) : 15-year follow-up from the survey study

by Quartel, Adrian

Published 2018

Other Authors: “...Jones, Simon A....”
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6

Clinical outcomes in a subpopulation of adults with Morquio A syndrome : results from a long-term extension study of elosulfase alfa

by Hughes, Derralynn A.

Published 2017

Other Authors: “...Jones, Simon A....”
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7

Evaluation of impact of anti-idursulfase antibodies during long-term idursulfase enzyme replacement therapy in mucopolysaccharidosis II patients

by Giugliani, Roberto

Published 2017

Other Authors: “...Jones, Simon A....”
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8

Genotype-phenotype relationships in mucopolysaccharidosistype I (MPS I) : insights from the International MPS I Registry

by Clarke, Lorne A.

Published 2019

Other Authors: “...Jones, Simon A....”
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9

Long-term safety and clinical outcomes of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency : two-year results

by Diaz, George A.

Published 2022

Other Authors: “...Jones, Simon A....”
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10

Sanfilippo syndrome : consensus guidelines for clinical care

by Muschol, Nicole

Published 2022

Other Authors: “...Jones, Simon A....”
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11

One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency

by Diaz, George A.

Published 2021

Other Authors: “...Jones, Simon A....”
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12

Impact of Elosulfase Alfa on Pain in Patients with Morquio A Syndrome over 52 Weeks: MOR-008: A Randomized, Double-Blind, Pilot Study

by Treadwell,Marsha

Published 2017

Other Authors: “...Jones,Simon A....”
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13

by Borgwardt,Line

Published 2018

Other Authors: “...Jones,Simon A....”
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14

Ten years of the Hunter Outcome Survey (HOS) : insights, achievements, and lessons learned from a global patient registry

by Muenzer, Joseph

Published 2017

Other Authors: “...Jones, Simon A....”
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15

The Morquio A Clinical Assessment Program: Baseline results illustrating progressive, multisystemic clinical impairments in Morquio A subjects

by Harmatz, Paul

Published 2013

Other Authors: “...Jones, Simon...”
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16

Longitudinal analysis of endurance and respiratory function from a natural history study of Morquio A syndrome

by Harmatz, Paul R.

Published 2015

Other Authors: “...Jones, Simon A....”
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17

Long-term outcomes of systemic therapies for Hurler syndrome : an international multicenter comparison

by Eisengart, Julie B.

Published 2018

Other Authors: “...Jones, Simon A....”
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18

Functional network resilience to pathology in presymptomatic genetic frontotemporal dementia

by Rittman, Timothy

Published 2019

Other Authors: “...Jones, Simon...”
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