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Dr Porter’s Natural History Trial in America – the Merits of AttendingOur son, William, was diagnosed with NPC just over 5 years ago at the age of 9 months. Ever since the diagnosis, we have been strong believers in the merits of developing a database containing relevant information about the progress of the disease in patients with NPC. Therefore we were very excited to read about the natural history trial by Dr Forbes Porter in America. (“Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, type C”)..... |
Research News
Brains for Brain - B4B
New European Task Force on Brain and Neurodegenerative Lysosomal Storage Disorders (LSDs)
The mission of Brains for Brain is to catalyse research in the field of neurodegeneration and lysosomal diseases. Brains for Brain is formed as an association of highly distinguished scientists and clinicians working together to create and coordinate effort towards the comprehension of the pathophysiology processes of neurological disorders occurring in lysosomal storage disorders. The implementation of knowledge of blood brain barrier function and the development of new molecular and or biochemical strategies to overcome the blood brain barrier and treat neurological disorders is a central part of the effort. The achievement of these targets will allow the development of new approaches for therapy of the neurological complications of LSDs patients.
The task force takes advantage of the expertise of the most distinguished European scientists, leaders in basic and applied neurotechnology and neurology, grouped together to create a co-ordinated effort toward the comprehension of pathophysiology processes of neurological disorders in LSDs, the implementation of knowledge on the blood brain barrier and the development of new molecular and or biochemical strategies to overcome the blood brain barrier to treat these central nervous system disorders.
The B4B nickname of the group has been chosen to acknowledge the effort of the 4 initial sponsors (ACTELION, BIOMARIN, GENZYME and SHIRE Human Genetic Therapies) without which this brainstorming panel could not have been created.
Writing in the July 2007 issue of Gauchers News, Dr David Begley, a founder member of the task force, explains: “The plan is to now co-ordinate a pan-European research effort and to apply for EU and other international funding. As part of this effort family and patient associations, together with clinicians and researchers, from throughout Europe will be brought together to lobby for more research leading to more effective treatment. By combining in a consortium; knowledge can be pooled and exchanged; the efforts of more research teams can be harnessed and co-ordinated; greater financial resource is available; progress can be accelerated to the ultimate benefit of more patients and families throughout Europe. Together more can be achieved’.
To read more about B4B visit www.brains4brain.eu