Future and perspectives

 

Innovative therapeutic strategy

LaboThis new therapeutic strategy addresses the clinical and molecular diversity of RDEB and DDEB patients. Besides transplantation of genetically corrected skin, it opens new fields in DEB therapeutic research leading to systemic or local delivery of small therapeutic molecules. It has a strong potential to bring clinical improvement to DEB patients and to represent a major progress in the treatment of this devastating disease.

 

 

 

By performing this research and development at the European level and in direct link with the European policy on rare diseases, results will serve as a model for the treatment of other severe genetic skin diseases as well as non-dermatological disorders. The results of the project will benefit the whole scientific community and will have positive impacts on the health of thousands of European citizens.

 

Effective treatment of RDEB will have also strong, positive knock-on effects on the quality of life of patients and their families. Restoring skin cohesion over the most vulnerable areas such as the hands and the feet to prevent the recurrence of skin lesions should result in a significant reduction in the functional and systemic complications of RDEB. Consequently, vital prognosis would improve, and the frequency and duration of hospitalization would be reduced. Further anticipated benefits include a reduction in pain and the prevention of deformity, with a positive effect as regards to the ability to carry out normal everyday activities. It is hoped, then, that physical improvements will result in lesser social handicap and better mental health. In addition, by cutting down the workload and stress involved in the care of the young patients, the positive impact of the therapy on their families would be considerable.

 

GENEGRAFT Project Development