Type of Vitamin E may be a treatment or cure for Niemann-Pick type C disease and other lysosomal diseases.
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Research News
FDA rejects Zavesca panel recommendation.
Unfortunately, in mid-March the FDA advised pharmaceutical company Actelion, which markets Zavesca that they would not yet approve Zavesca for NPC; that more definitive proof is required by the law. We are hopeful that further study efforts will lead to future approval. The consequence of the FDA’s decision is that too many children with NPC …