The MEBO Blog: first gene therapy drug in Western world gets market approval

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Denver TMAU Test Lab survey click here

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USA survey for anyone who wants to improve Denver TMAU test

begun : Dec22
end : no ending for now

A trainee genetic counselor is working at the Denver TMAU test lab. Probably as part of her training. As a project she wishes feedback on any aspect of the Denver TMAU test and process. You can fill in the survey and/or email her (email address is in survey). It's meant for USA people, but perhaps others can give their view too (as we have so few opportunities).

quote from her rareconnect post

"Hello all! I wanted to make you aware of a research study being conducted to better understand the experience and needs of individuals with trimethylaminuria with a goal of being able to create improved patient and healthcare provider education materials. Any participation is completely voluntary and all responses remain confidential. Feel free to use the contact information within the link with any questions or share the survey with others with TMAU."

see this post for more details

https://www.meboblog.com/2023/01/denver-tmau-test-survey-tbc-who-it-is.html

Monday, November 5, 2012

first gene therapy drug in Western world gets market approval

The first gene therapy drug in the western world has been granted a licence in the European Union (EU) and is therefore approved for the drug market in the EU.

Glybera, a gene therapy drug which has been used in trials for the disorder lipoprotein lipase deficiency, is made by Dutch company UniQure. In theory 'gene therapy' could prove to be 'cures' for genetic disorders, or at least improve the function of the gene at fault. Up until now worldwide only 2 gene therapy drugs have been approved, both in China.

In relation to genetic malodor disorders, obviously gene therapy for the FMO3 gene would be a candidate.

A EURORDIS and
NORD Member Organization

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Monday, November 5, 2012

first gene therapy drug in Western world gets market approval