It has been a banner year in Neuronal Ceroid Lipofuscinosis (NCL) research. Clinician scientists and investigators have traveled the globe presenting their findings at scientific meetings and...
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Other Batten Disease Research
Waste management is a big issue anywhere, but at the cellular level it can be a matter of life and death. A...
In Summer 2010 Rare Disease UK (RDUK) carried out a survey of patients and families affected by rare diseases. The aim of this survey was to find out more about the experiences of people living...
This trial aims to establish the safety and tolerability of short-term (8 weeks) administration of mycophenolate mofetil (Cellcept) in ambulatory children with JNCL. It also aims to gather...
The Cornell University gene therapy trial for Late Infantile Batten Disease has started recruiting children. The trial is based at Weill Medical College, Cornell University under the...
Dr Ruth Williams and Dr Sara Mole in this alogorithm outline the paths of diagnosis for NCL based on clinical experience and recent research findings. A number of experts were consulted and...
The symptom profile and experience of children with rare life-limiting conditions: Perspectives of their families and key health professionals.
C Malcolm, S Adams, G Anderson, F Gibson...
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