AGU rare disease

27/03/2023

First part of the betaine study is published!

Novel treatment strategies are emerging for rare, genetic diseases, resulting in clinical trials that require adequate biomarkers for the assessment of the treatment effect. For enzyme defects, biomarkers that can be assessed from patient serum, such as enzyme activity, are highly useful, but the ac...

29/11/2022

https://www.gofundme.com/f/agu-cure

Parents of Daniel and Alexander, Sydney and George, Hermance, Makeda, Trisha, Selman, Jor and half … Julia Taravella needs your support for Hope for AGU Cure

18/10/2022

Today is the first day of the last study for AGU gene therapy. With this study we will determine the dose of the drug and prove that the drug that will be given to AGU children is not toxic. If everything goes as planned technically and finances are secured, we will receive FDA permission to start the clinical trial in six months!

20/04/2022

Progress in AGU treatment!

01/12/2021

It's Giving Tuesday!

https://www.gofundme.com/f/agu-cure?viewupdates=1&rcid=r01-163828824239-530294fe95774412&utm_medium=email&utm_source=customer&utm_campaign=p_email%2B1137-update-supporters-v5b

29/10/2021

Gene therapy is still quite a new concept to many people. The first ideas to replace the gene was published in 1970s. A few years later, in 1990, a 4-year-old girl with a severe combined immunodeficiency (SCID) became the first gene therapy success story. It became possible to treat many thousands of genetic diseases and can give hope of life for fatal or debilitating diseases. As of today, there are several approved gene replacement therapies in US and Europe.

We are working to bring such a treatment for AGU, a rare fatal disease with no treatment. We contracted several companies to make gene therapy medicine. You can see the production steps in the picture attached. It will take about a year to complete. We stared with the step 1 (production of the AGA gene) in October 2021.

04/10/2021

Interesting article about Spanish royal family - Carlos II of Spain (1661–1700) had AGU?

Carlos II of Spain (1661–1700), last of the Spanish Habsburgs, was known as The ‘Bewitched’ due to his multiple medical issues and feeble nature. He suffered from a range of ailments extending beyond the well-known Habsburg jaw, including developmental delay, intellectual disability, dysarthri...

22/09/2021

Fundraising for saving Trisha and other AGU kids!


https://www.gofundme.com/f/helpsavetrisha

25/07/2021

Please, help AGU- kids live longer! Donations needed as clinical trials are approaching.

Raretrait.com needs to raise $2,000,000 to pay for the medicine that will be used in a clinical trial next year in the United States.
Please, help AGU-kids to live longer!

Auta AGU-lapsia elämään pidempään! Rahalahjoituksia kaivataan, sillä AGU-sairauden geeniterapian kliiniset testit lähestyvät.
Raretrait.com yrittää kerätä vielä 2 miljoonaa dollaria rahoitusta maksaakseen lääkkeen valmistamiskustannuksia ja potilaiden hoitomaksuja, kun kliiniset testin ensi vuonna olisivat valmiita alkamaan USA:ssa.

Voit tehdä lahjoituksen gofundme-linkin kautta. Kaikki summat tervetulleita!

https://www.gofundme.com/f/agu-cure?utm_source=widget&utm_medium=referral&utm_campaign=p_cp+share-sheet

Parents of Daniel and Alexander, Sydney and George, Hermance, Makeda, Trisha, Selman, Jor and half … Julia Taravella needs your support for Hope for AGU Cure