Aims and Objectives

In order to advance the development of treatment options for the NCL diseases, the DEM-CHILD project aims at
(1) increasing knowledge of biological processes and pathomechanisms involved in the NCL diseases and their impact on the clinical spectrum of these diseases

(2) at transposing this knowledge into clinical applications for prevention and early detection of these diseases

(3) at ensuring that clinical (including epidemiological) data guide further research towards the establishment and evaluation of innovative therapies.

For this purpose, the project will combine the expertise of (i) recognized European research teams, both basic scientists and clinicians, (ii) high-technology SMEs, (iii) experts in medical ethics, and (iv) NCL patients and family associations.
The objectives of this multidisciplinary approach to study NCL diseases in Europe and India are
(1) development of new testing and screening methods applicable to the wider community
(2) prevention and early detection of NCL diseases,
(3) studying disease prevalence
(4) quantitative description of natural histories
(5) characterisation of molecular basis and pathophysiology in relevant models
(6) development of innovative therapies for these diseases.