Next steps to finding a cure for Alternating Hemiplegia of Childhood
At the DIA Euromeeting I met some very interesting people, as an example a couple from UK that represent Alström Syndrome.They have been very active for the last 15 years and lost 2 children to this rare disease (1 in a million)
The disease was given a grant for 1.5 million Pounds for a project regarding Alström but there will be no outcome of that project because the experts did not produce what was promised.
The gene that causes Alström was discovered 7 years ago, they worked with mouse models but still have not found a drug that works for the disease.
They also had the problem of giving all their data to the experts that got the grant and then those experts denied them access to the data. That delayed their progress because that had to be settled in court which the Alström syndrome UK won.
AHC needs to have a European platform (that we now have with AHCFE) to fully participate at a European level.
EURORDIS has been doing a great job and they are seen as THE patient advocates in Europe. The CEO of Eurordis was awarded for excellent work as patient advocates in Europe.
It is clear that the patient organizations are becoming more and more active and will have more influence on all aspects regarding diseases and we need to make the most of this trend.
We live in transitional times where experts in medicine, research and industry are realizing that spending more time listening to patients is extremely helpful for their research
This relationship can cut research time and is very helpful when getting patients to register for clinical trials = easy access to patient via patient organization and bio banks
Each time the industry invests in the relationship with patent organizations the return will be multiplied and of course this relationship needs to be beneficial for both parties
SJ
