Morquio B

Our future plans

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Our goal is to bring together experts in pediatrics, genetics, neurology, radiology, orthopaedic surgery and biochemistry to work as a team to investigate how we can better understand and treat those born with mutations in the GLB1 gene leading to the development of Morquio B and late-onset GM-1 syndromes. Our first priority is to overcome the research constraint of the low numbers associated with rare diseases by populating our database with as many professionals and afflicted individuals as possible.
We intend to use the MB Registry professional and patient databases with the aim to foster more directed and coordinated efforts to provide accurate and timely diagnosis of these conditions, and to get a better understanding of long term outcomes and quality of life of these patients. Our database will serve as a reliable source of potential trial candidates when any new treatments are proposed such as small molecule, enzyme replacement or gene therapy.
In addition, we envisage clinicians and research groups being able to leverage their strengths and finding suitable complementary partners for the purpose of engaging in collaborative research, to find cures for these conditions, including efforts to secure funding and support for these endeavours.
The MB Registry will be regularly revised to reflect updates in diagnosis, new insights into pathophysiology, present and new treatments, as well as fostering the use of social media for the benefit of patients, families and health care researchers and providers.

Morquio B Disease

About this initiative

This project has been initiated by The Priest Family of Vancouver BC Canada, who have a 8 year old son with Morquio B disease. Their donation to the Canadian MPS Society has made this Morquio B registry possible. The projected is supported by TIDE BC infrastructure funds and the University of British Columbia.

Part of TIDE BC

The mission of TIDE BC, a care and research initiative, is to create an evidence-based Best Care Practice to enhance diagnosis and treatment of rare metabolic diseases. We all are motivated by the concept of treatable inborn errors of metabolism, the possibility to translate research into better care, striving to ensure that every child has access to early diagnosis and the best available treatment. International collaboration and patient & family participation are the pillars of our work. For more information on the TIDE BC project, please visit our website.