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WORLD Symposium Notebook: New Data In Lysosomal Diseases And A Push For Earlier Testing

 
• By Mandy Jackson

The WORLD Symposium Feb. 13 to 16 in San Diego offered everything from basic research through late-stage clinical data in lysosomal diseases, including ArmaGen's blood-brain barrier-crossing treatment for Hurler syndrome and updates from ongoing development programs from rare disease specialists, such as Genzyme, Amicus and Protalix.

Hands

The WORLD Symposium from Feb. 13 to 16 in San Diego brought together academic researchers and biopharmaceutical companies to share emerging research in lysosomal diseases, including next-generation enzyme replacement therapies, gene therapies and tests that identify patients before debilitating effects of the diseases kick in.

Researchers, clinicians and biopharma company scientists presented a range of basic research, preclinical data and clinical trial results for Gaucher, Fabry and Farber diseases, Hunter and Hurler syndromes, and other rare and ultra-rare lysosomal diseases. ArmaGen Technologies Inc., Shire PLC, Sanofi's Genzyme business unit, BioMarin Pharmaceutical Inc. and Amicus Therapeutics Inc

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