The Rare Disease Registry Program (Gaucher, Fabry, Pompe, MPS1) Amd 11 - RENEWAL
Research type
Research Database
IRAS ID
341912
Contact name
Simon Jones
Contact email
Research summary
The Rare Disease Registry Program (The X-Registries)
REC name
North West - Greater Manchester Central Research Ethics Committee
REC reference
24/NW/0102
Date of REC Opinion
3 Jul 2024
REC opinion
Further Information Favourable Opinion
Data collection arrangements
This is a long term ongoing non-interventional observational program (there is currently no end date) As an observational database it tracks the natural history and outcomes of patients with Pompe, Fabry, Gaucher and MPS1 Disease. The primary purpose of the Registry program is to collect uniform and meaningful clinical data on patients with rare disease.
Each Registry Program is specifically centered around that particular disease and is focused on collecting key standard of care assessments and clinical outcomes associated with that diseases management.Research programme
The objectives of the Registry Program are to enhance the understanding of the variability, progression, and natural history of these diseases with the ultimate goal of better guiding and assessing therapeutic intervention; to assist the medical community treating patients with these diseases with recommendations for monitoring patients and reports on patient outcomes to optimize patient care; to characterize disease populations and to evaluate the long-term safety and effectiveness of Enzyme Replacement Therapy (ERT). Data from the Registry are also used to fulfil various global regulatory commitments, to support product development/reimbursement, and for other research and non-research-related purposes.
Research database title
The Rare Disease Registry Program (The X-Registries)
Establishment organisation
Sanofi B.V.
Establishment organisation address
Paasheuvelweg 25
Amsterdam
NETHERLANDS
1105 BP