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March20 podcast Dr Hazen
anti-TMA pill in a year or 2 ? (scroll 12 mins)

Additional info: https://youtu.be/811v7RLXP9M
MEBO Karen
at UK Findacure conf 2020

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MEBO TMAU TESTING DISCONTINUED
(2012-2017)

MEBO Map Testing & Meetups


Full details : https://goo.gl/TMw8xu
want listed ? contact info@meboresearch.org

MEBO - UBIOME study 2018

'PRESS RELEASE'

NCT03582826
ClinicalTrials.gov

MEBO Gut Microbiome Study
"Microbial Basis of Systemic Malodor and PATM Conditions (PATM)"
Funded by uBiome Research Grant

"Microbial Basis of Systemic Malodor and PATM Conditions (PATM)"

Dynamics of the Gut Microbiota in
Idiopathic Malodor Production
& PATM

Started May 2018 - Ongoing

Current people sent kits : 100/100
3 kits per person

NO LONGER RECRUITING

Participation info : LINK English

MEBO Private Facebook Group
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BO Sufferers Podcasts

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Petitions

TMAU Petition world
TMAU UK end total:262
TMAU UK ends 23/01/20
TMAU Petition USA end total 204
USA : Moveon open
TMAU (Dominican)
Metabolomic Profiling Study
NCT02683876

Start : Aug 2016
Stage 1 : 27 Canadian volunteers to test
Latest click here (26 oct) :
17 samples returned


Note : Stage 1 is Canada only.
Return cut-off date : passed
Analysis can take 6/8 weeks
Analysis start in/before Nov
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NORD Member Organization
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MEBO survey for Dr Hazen click here
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Regular readers will know that Dr Stan Hazen et al at Cleveland Clinic are developing a TMA-blocker pill, as they proposed in a 2011 paper that TMAO is a factor in CVD. Recently Dr Hazen and colleagues contacted MEBO as they have always thought they could also help with TMAU. This survey is to give them an idea of the 'state of the community'. It is a "version 1". They may not even look (though they have access permission), but it could be useful to give them an overview of the community

MEBO had a zoom call with Dr Hazen and his team in October. Another zoom call is planned when they have time

MEBO Research Clinical Trials

Friday, July 27, 2012

First western-world 'gene therapy' drug receives approval in Europe

Quote from UniQure press release

UniQure announced today that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion that recommends marketing authorization of Glybera® (alipogene tiparvovec) as a treatment for lipoprotein lipase deficiency (LPLD) under exceptional circumstances.

gene therapy for genetic TMAU ?
Gene Therapy has been seen as a Holy Grail cure for metabolic disorders, especially disorders where a single enzyme is at fault. However since gene therapy stated to be researched between 10-20 years ago, not one drug has received approval in the western world by the appropriate regulatory bodies. Finally, a gene therapy drug has received approval in Europe, to become the first approved gene therapy drug in the western world.

The drug, Glybera, is for the rare metabolic disorder lipoprotein lipase deficiency, a disorder that currently has no alternative treatment.

The drug has been developed by UniQure in the Netherlands. The website says gene therapy is particularly suitable for single enzyme disorders, which would be the case for FMO3 deficiency and probably any other metabolic malodor disorders that have not been recorded yet.As well as already progressing in some trials for other rare metabolic disorders, they also expect it can treat other disorders such as Parkinson's disease and hearing loss, which UniQure are already investigating.

Below are some quotes regarding Glybera from the Uniqure press release

"First gene therapy in the Western world to reach important regulatory approval milestone, culminating 40 years of research"

"Validates uniQure’s unique AAV-based gene therapy platform, consisting of a modular, plug-and-play vector system and unrivaled GMP manufacturing capabilities on a commercial scale"

"After today’s positive recommendation, Glybera is poised to become the first in a class of gene therapy products approved in Europe to treat orphan diseases, rare conditions with a very high unmet medical need.” Marketing authorization covers all 27 European Union member states."

"Now, for the first time, a treatment exists for these patients that not only reduces this risk of getting severely sick, but also has a multi-year beneficial effect after just a single injection. The positive recommendation from the CHMP for Glybera therefore represents a major breakthrough for both LPLD patients and for medicine as a whole. Restoring the body's natural ability to break down fat particles in the blood in order to prevent pancreatitis and excruciating abdominal pain suffered by patients, is what gene therapy is all about: curing disease at the genetic level."

"At uniQure we are developing treatments for a number of other rare diseases as well, such as acute intermittent porphyria and Sanfilippo B. But the potential of gene therapy stretches far beyond rare diseases. As shown recently in a publication in the New England Journal of Medicine (N Engl J Med 2011; 365:2357-2365, December 22, 2011), hemophilia patients treated with our proprietary gene are showing a sustained clinical effect over several years, which has allowed prophylaxis treatment to be stopped. In addition, we are advancing programs in degenerative diseases such as Parkinson’s. We believe that just like antibodies, gene therapy will one day be a mainstay in clinical practice"

"The Company is now preparing to apply for regulatory approval in the US, Canada, and other markets."

"Data from these clinical trials indicate that a single dose administration of Glybera resulted in a long-term biological activity of the LPL protein."

Press Release on UniQure website

Links
NY Times article on Glybera being first western-world gene therapy drug to get approval

Glybera article in UK newspaper The Guardian

Gene therapy nears approval in Europe, BBC News Health

Gene therapy and stem cells unite, BBC News Health

1 comments:

Anonymous said...

more media links =)
http://www.bbc.co.uk/news/health-18926892

http://www.bbc.co.uk/news/health-15272081

Jul 29, 2012, 3:34:00 PM
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