Morquio B
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  • Morquio B Disease mutations are on the same gene as GM1 Gangliosidosis (GM1G). [+]

    Yet, GM1G leads to neurodegeneration and intellectual disability whereas Morquio B Disease does not appear to. Why are the responses different and is there any overlap?

  • Morquio B Disease is predominately a bone disease however, it is extremely rare. [+]

    The number of patients identified in the past 30 years are very few and we still cannot say with complete confidence if some adults classified as Morquio B Disease in childhood started to exhibit gene-related symptoms of neurodegeneration later in life in a manner similar to GM1G.

  • New treatment strategies for some GLB1 mutations have been developed. [+]

    So far these are in laboratory models, however, will these same results be seen in patients, and will it benefit people with Morquio B Disease?

  • The promise of new treatments is not enough [+]

    To turn promise into prescription we have to engage in research that leverages the strengths and experiences of multiple clinicians and researchers. There are already early-stage discoveries that are in cell and animal model stage. We need to have a sufficient number of patients, and a complete understanding of their condition as individuals and as a group, if we are to achieve clinical trial status for any of these promising therapies.

  • It’s all in the numbers… [+]

    At a local level, researching Rare Diseases is almost impossible due to lack of numbers. When approached globally, the problem of numbers becomes less of an issue. By exploiting internet technologies, we can get more patients and committed health care and research professionals involved. Our Morquio B Registry is the foundation of bringing together all these people.

What can a Morquio B Registry Accomplish?

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By bringing together interested parties, including those involved with late-onset GM-1, we can identify who we are, where we are and what are we doing.
Collectively we can bring enough clinicians, researchers and patients together to better understand the Morquio B condition, its progress and its treatment.
By being online, we can dynamically respond and adapt to the needs of the Morquio B community to as more people learn about us and register with us.

It all sounds good but, will it work?

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The Morquio B Registry is modelled on success. Our registry is under the direction of the TIDE BC Initiative.
This initiative has a proven track record of elucidating characteristics of rare diseases that allow them to be better understood, even to the point of having found a treatment for some, where no treatment was considered possible.

Morquio B Disease mutations are on the same gene as GM1 Gangliosidosis (GM1G).

Yet, GM1G leads to neurodegeneration and intellectual disability whereas Morquio B Disease does not appear to. Why are the responses different and is there any overlap?

Morquio B Disease is predominately a bone disease however, it is extremely rare.

The number of patients identified in the past 30 years are very few and we still cannot say with complete confidence if some adults classified as Morquio B Disease in childhood started to exhibit gene-related symptoms of neurodegeneration later in life in a manner similar to GM1G.

New treatment strategies for some GLB1 mutations have been developed.

So far these are in laboratory models, however, will these same results be seen in patients, and will it benefit people with Morquio B Disease?

The promise of new treatments is not enough.

To turn promise into prescription we have to engage in research that leverages the strengths and experiences of multiple clinicians and researchers. There are already early-stage discoveries that are in cell and animal model stage. We need to have a sufficient number of patients, and a complete understanding of their condition as individuals and as a group, if we are to achieve clinical trial status for any of these promising therapies.

It’s all in the numbers…

At a local level, researching Rare Diseases is almost impossible due to lack of numbers. When approached globally, the problem of numbers becomes less of an issue. By exploiting internet technologies, we can get more patients and committed health care and research professionals involved. Our Morquio B Registry is the foundation of bringing together all these people.

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

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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.
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