Christine A. Lavery
Amersham, ENG, United Kingdom
Last Updated: 09/26/2026
Save publications for later
Sign Up
Not sure about your diagnosis?
Check Your Symptoms
Found 9 publications
The patient's view on rare disease trial design - a qualitative study.
Journal: Orphanet journal of rare diseases
Published: March 07, 2018
Mortality in patients with Sanfilippo syndrome.
Journal: Orphanet journal of rare diseases
Published: July 25, 2017
Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III.
Journal: Orphanet journal of rare diseases
Published: March 29, 2017
The diagnostic journey of patients with mucopolysaccharidosis I: A real-world survey of patient and physician experiences.
Journal: Molecular genetics and metabolism reports
Published: June 22, 2016
Long-Term Cognitive and Functional Outcomes in Children with Mucopolysaccharidosis (MPS)-IH (Hurler Syndrome) Treated with Hematopoietic Cell Transplantation.
Journal: JIMD reports
Published: June 26, 2015
Recommendations for initiation and cessation of enzyme replacement therapy in patients with Fabry disease: the European Fabry Working Group consensus document.
Journal: Orphanet journal of rare diseases
Published: January 15, 2015
Burden of disease in patients with Morquio A syndrome: results from an international patient-reported outcomes survey.
Journal: Orphanet journal of rare diseases
Published: December 17, 2013
Pseudotyping the adenovirus serotype 5 capsid with both the fibre and penton of serotype 35 enhances vascular smooth muscle cell transduction.
Journal: Gene therapy
Published: January 17, 2013
Showing 1-9 of 9
Last Updated: 09/26/2026