Somebody asked me the other day if I really think we can SMASH SMA or if its just a campaign to raise attention about the disease that is the number one genetic killer of children - a disease that is carried by 1 in 40 of us.
I hope this blog gives the answer
Those of you who are regular readers will be familiar with the work of Prof. Gillingwater and his team in Edinburgh. They are leading research on finding solutions to SMA and we are backing them by encouraging awareness and funding for them.
I had the following mail from Tom today
Hi Tom & Maria,
I hope you are both keeping well.
I wanted to update you on some very exciting progress in our SMA research that has come through over the last couple of weeks:
- First, we have had a huge number of high-quality applications for the Estella Star SMA Scholarship. We are going to be interviewing over the next couple of weeks and will let you know who we have appointed ASAP. We will ask them to write a short report for you when they finish their project to let you know how they got on.
- Second, we have made some really exciting progress with identifying a potential new drug for SMA. We are going to use the remainder of the funds you have helped raise to undertake pre-clinical testing in SMA mice. Any additional funds that come in will also be used for this trial (we are also hoping to approach some of the large funding bodies in an attempt to raise some serious cash for this...we need at least £50k!). If the results of this trial are positive, and show a beneficial effect of the drug in the SMA model mice, we would hope to try and move the drug towards human clinical trials as quickly as possible.
Both of these developments would not have been possible without your incredible advocacy and support. I can't begin to thank you both enough. I hope that you feel that these developments are moving things in the right direction and that together we can SMASH SMA!
Very best wishes,
They have made some really exciting progress with identifying a potential new drug for SMA and they are going to use the funds you donated to trial this on mice.
The really exciting news though is that a donation of £ 50,000 will enable them to move the drug towards human clinical trials.
That is earth shattering
There are millions of pounds donated to charities and pharmacutical firms to help solve SMA and here is the team who may have the solution and they are based in the UK. That has to be worth a 50k gamble.
We will now dedicate the fund rasing side of SMASHSMA to trying to raise the 50k that Tom and his team need. Every donation counts.
Here is the web link to make a donation if you would be so kind.
The idea of there being a scholorship in Estella's name is moving beyond words.
Am i serious about SMASHING SMA ?
It's what I promised her.
We are going to cure this disease.