Distinguished in Sandhoff Disease

Simon A. Jones

Manchester University NHS FT, 
Manchester, ENG, GB 
Distinguished in Sandhoff Disease
Manchester University NHS FT, 
Manchester, ENG, GB 
OverviewLocationsClinical Research

Overview

Simon Jones practices in Manchester, United Kingdom. Mr. Jones is rated as a Distinguished expert by MediFind in the treatment of Sandhoff Disease. His top areas of expertise are Cholesteryl Ester Storage Disease, Mucopolysaccharidoses (MPS), Wolman Disease, Lysosomal Acid Lipase Deficiency, and Bone Marrow Transplant.

His clinical research consists of co-authoring 231 peer reviewed articles and participating in 8 clinical trials. MediFind looks at clinical research from the past 15 years. In particular, he has co-authored 2 articles and participated in 2 clinical trials in the study of Sandhoff Disease.

Gender
Male

Locations

Manchester University NHS FT, Manchester, ENG, United Kingdom
Other Locations
Oxford Road, Manchester, ENG, United Kingdom

Clinical Research

Clinical research consists of overseeing clinical studies of patients undergoing new treatments and therapies, and publishing articles in peer reviewed medical journals. Experts who actively participate in clinical research are generally at the forefront of the fields and aware of the most up-to-date advances in treatments for their patients.

8 Clinical Trials

A Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, Safety, and Tolerability of Venglustat in Late-onset GM2 Gangliosidosis (Tay-Sachs Disease and Sandhoff Disease) Together With a Separate Basket for Juvenile/Adolescent Late-onset GM2 Gangliosidosis and Ultra-rare Diseases Within the Same and Similar Glucosylceramide-based Sphingolipid Pathway
A Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, Safety, and Tolerability of Venglustat in Late-onset GM2 Gangliosidosis (Tay-Sachs Disease and Sandhoff Disease) Together With a Separate Basket for Juvenile/Adolescent Late-onset GM2 Gangliosidosis and Ultra-rare Diseases Within the Same and Similar Glucosylceramide-based Sphingolipid Pathway
Enrollment Status: Terminated
Publish Date: December 30, 2024
Intervention Type: Drug
Study Drug: Venglustat GZ402671
Study Phase: Phase 3
Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease): A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study
Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease): A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study
Enrollment Status: Completed
Publish Date: March 12, 2024
Intervention Type: Drug
Study Drug: IB1001
Study Phase: Phase 2
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Enrollment Status: Completed
Publish Date: March 05, 2024
Intervention Type: Drug
Effects of N-Acetyl-L-Leucine on Niemann Pick Type C Disease: A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study.
Effects of N-Acetyl-L-Leucine on Niemann Pick Type C Disease: A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study.
Enrollment Status: Completed
Publish Date: November 28, 2023
Intervention Type: Drug
Study Phase: Phase 2
An Open-Label Adaptive-Design Study of Intracisternal Adenoassociated Viral Vector Serotype rh.10 Carrying the Human β-Galactosidase cDNA for Treatment of GM1 Gangliosidosis
An Open-Label Adaptive-Design Study of Intracisternal Adenoassociated Viral Vector Serotype rh.10 Carrying the Human β-Galactosidase cDNA for Treatment of GM1 Gangliosidosis
Enrollment Status: Terminated
Publish Date: June 09, 2023
Intervention Type: Genetic
Study Phase: Phase 1/Phase 2
Study of Peri-Articular Anaesthetic for Replacement of the Knee
Study of Peri-Articular Anaesthetic for Replacement of the Knee
Enrollment Status: Completed
Publish Date: November 04, 2022
Intervention Type: Drug
Study Drugs: Liposomal Bupivacaine, Bupivacaine
Study Phase: Phase 3
A Retrospective and Cross-sectional Study to Evaluate Neurodevelopmental Status in Pediatric Subjects With Severe Mucopolysaccharidosis Type II (Hunter Syndrome)
A Retrospective and Cross-sectional Study to Evaluate Neurodevelopmental Status in Pediatric Subjects With Severe Mucopolysaccharidosis Type II (Hunter Syndrome)
Enrollment Status: Completed
Publish Date: April 26, 2022
A Prospective and Retrospective Data Collection Study to Evaluate Outcomes in Males ≤17 Years of Age Undergoing Allogeneic Hematopoietic Stem Cell Transplantation for the Treatment of Cerebral Adrenoleukodystrophy
A Prospective and Retrospective Data Collection Study to Evaluate Outcomes in Males ≤17 Years of Age Undergoing Allogeneic Hematopoietic Stem Cell Transplantation for the Treatment of Cerebral Adrenoleukodystrophy
Enrollment Status: Terminated
Publish Date: May 21, 2020
Intervention Type: Genetic
View 7 Less Clinical Trials

230 Total Publications

Unmet needs in the treatment and care of somatic manifestations in mucopolysaccharidosis type II: A targeted literature review.
Unmet needs in the treatment and care of somatic manifestations in mucopolysaccharidosis type II: A targeted literature review.
Journal: Molecular genetics and metabolism
Published: August 05, 2025

Areas of Expertise

MediFind evaluates expertise by pulling from factors such as number of articles a doctor has published in medical journals, participation in clinical trials, speaking at industry conferences, prescribing and referral patterns, and strength of connections with other experts in their field.

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