The advent of effective enzyme therapies for the visceral manifestations of Gaucher disease has stimulated renewed interest in both the basic science and therapeutics for the neuronopathic Gaucher disease variants, types 2 and 3. In numerous Scientific workshops, led by Dr. Tony Futerman and hosted by the PBRF, basic scientists discussed the mechanisms causing the brain disease in Gaucher disease. The discussion was vigorous and will lead to many interesting and important discoveries related to the neurologic involvement in Gaucher disease. Continue reading Development Plan FDA and EMA Approval
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