832 signatures for gene therapy

European researchers have introduced a European grant proposal in order to raise funds for a clinical trial to cure PKD through gene therapy. 

The consortium team behind this proposal would like to thank everyone who signed the letter of support. The fact that up to 832 persons from 27 different countries, affected directly or indirectly by PKD gave their support, is certainly going to be very important for the potential success of the proposal. This is how this is stated in the grant proposal:

“Although the absolute number of patients with a rare disease is  low, the devastating severe effects of the disease and the poor quality of life of the patients have a huge social impact on parents, relatives and friends. This is underscored and exemplified by the response the initiative of ForGeTPKD has received. In less than 3 weeks after posting the ForGeTPKD initiative on the website of the International PKD patient Support Group (march 30, 2015) the project received the support of 832 people directly involved in or connected to pyruvate kinase deficient patients.

Link to PKD # of signatures
I’m a family member of someone with PKD 131
I’m a friend of someone with PKD 421
I’m a parent of a child/adult with PKD 48
I’m a patient with PKD 37
Other 195

Given these numbers, pyruvate kinase deficiency has a huge social impact, and affects in this way the lifes of at least 25 times more people than the absolute number of people directly affected by the disease.

The signatures came from around the world (27 countries!), but most people who signed the letter came from Spain, USA, France, The Netherlands, Denmark, Mexico, Australia, Belgium, UK and Canada.

The proposal was submitted on April 21st and there is a 6 months waiting period for the results. Only ~10% of the 130 presented proposals at the stage 2 will be funded. The researchers already passed the first stage were a 25% rate success was applied (accepting 130 out of 421 presented proposals).

Let’s cross our fingers this proposal will be accepted!

Leave a Reply

Fill in your details below or click an icon to log in:

WordPress.com Logo

You are commenting using your WordPress.com account. Log Out /  Change )

Facebook photo

You are commenting using your Facebook account. Log Out /  Change )

Connecting to %s