Lisa Flint

AADC Gene Therapy Appeal - Climbing a Mountain of Promise! Phase 2

Fundraising for The AADC Research Trust
£13,408
raised of £40,000 target
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AADC Families - Gene Therapy Appeal!, 3 April 2012
The AADC Research Trust

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RCN 1114367
We fund AADC medical & scientific research to improve the lives of our sick children

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Phase 2 – Active (Feb 2013)

WE NEED TO NOW RAISE £40,000 ($60,000)

With your help we raised an amazing amount of money from our ‘AADC Gene Therapy Appeal - A Ray of Sunshine and HOPE’ (Phase 1) … £38,000…THANK YOU!!!

From funds raised in Phase 1 of the appeal the PND Association and The AADC Research Trust were able to afford the $50,000 (£33,000) required by Professor Bankiewicz team to complete the regulatory documents relating to the AADC Gene Therapy Trial.  Happily we can report these documents have now been completed and we are awaiting the final FDA seal of approval following toxicology studies before being able to set a firm date for this trial.

Unfortunately we have a considerable amount of work still to do and firstly we must report the disappointing news that the trial has been pushed back to early 2014 and we must now work hard and raise a further $60,000 (£40,000). 

This is to fund an experienced neurological medical professional to dedicate time to the preparation of pre and post AADC patient selection protocols. These include determining the exact inclusion criteria, the logistics of how patients will be screened and selected.  We need to figure out each step of the trial – what evaluations need to take place, where and who does them, what complications could arise and how they will be managed etc… Ultimately the safety of our vulnerable children before, during and after these trials is paramount.

In addition to Phase 1 and 2, Phase 3 will see us trying to raise a further $23,000 (£15,000) in order to organise a patient and professional AADC Gene Therapy workshop in both the USA and UK…this will enable families to travel and learn about every aspect of this procedure before making the incredibly difficult decision to include their child in the trial. This trial has the potential to not only drastically change the lives of our children but to also set the bar for future gene therapy treatment across all rare neurotransmitter brain diseases…

I urge you to dig deep and help make this happen and on behalf of these very sick children I am asking you to find some way to help us raise these funds as quickly as possible.

However small your contribution is it will still undoubtedly have a massive impact on the future of our AADC deficient children…

Once again on behalf of all our children… Thank you!

Fundraising

Join us for the British 10k London Run (July 2013) or make up a team for the Bear Grills Nuts Challenge & BBQ (August 2013) – contact emma@aadcresearch.org for more details.

Create your own page and ‘Bee Different’ by doing it for AADC

Phase 1 completed

This is an INTERNATIONAL plea on behalf of every familiy around the World whose child is suffering with AADC deficiency.

Aromatic Amino Acid Decarboxylase (AADC) deficiency is a rare, multiple disabling and life threatening brain disease...

AADC Gene Therapy is a treatment which is just around the corner and we desperately need your help to bring it to our suffering AADC deficient children throughout the WORLD!

AAV-AADC was originally a gene vector created for Parkinson ’s disease by Professor Krystof Bankiewicz (Prof. B) in the USA...but as luck would have it, this is also a treatment potentially suitable for AADC deficient children too. It has the capability to be life-changing and lead to impressive improvements even to the most disabled of our children.

We are raising funds to help Prof. B complete all the necessary safety regulatory requirements in order to bring this trial to life...

The first Gene Therapy trial will be at the NIH in the USA and hopefully commence mid-year 2013.

Our target to raise is massive $50,000. But this is a fraction of the overall cost of the trial. The AADC Research Trust has already set aside $10,000 of its funds to this project and the PND and ALADD Foundation have still to announce their contributions.

PLEASE PLEASE give generously!

A GIGANTIC thank you from all our beautiful suffering children: Sydney, Delaini, Swathi, Jillian, Marlie, Luis, Gavin, Sydney Claire, Riley, Marissa, Jake, Ted, Marcus, Mohanad, Muiad, Vincent, Romain, Zain, Haia, Nathan, Sofia, Satoshi, Lily, Lavinia, Lynn, Joyce, Tehila, Ryder, Daniel, Aniya, Romanja, Hulya, Aimar, Peeter, Sellina and Emily

About our childrens disease...

AADC is a genetic disorders that affect the synthesis, metabolism and catabolism of neurotransmitters in children.

These inborn errors of metabolism affect the central nervous system in children and if left untreated can lead to severely compromised neurological function.

Visit: www.aadcresearch.org to learn more about this devastating disorder.

Contact: lisa@aadcresearch.org for more information about how you can help us raise funds!

Thank you for taking the time to visit our JustGiving page.

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So please dig deep and donate now...

THANK YOU!!!

 

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About the charity

The AADC Research Trust

Verified by JustGiving

RCN 1114367
AADC deficiency is a rare disabling brain disease. More than 100 children in 30 countries have been identified with the disease. It's proved fatal for some of them. The AADC Research Trust is a children's charity funding research, supporting affected families and promoting global disease awareness.

Donation summary

Total raised
£13,407.93
+ £528.16 Gift Aid
Online donations
£3,284.93
Offline donations
£10,123.00

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