Lysogene Recieves US Orphan Designation for its Lead Intracerebral Gene Therapy Product SAF-301

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LYSOGENE announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its lead gene therapy product SAF-301 for the treatment of Sanfilippo type A syndrome (also named mucopolysaccharidosis type IIIA), a rare, genetic and life-threatening disease affecting children (http://www.accessdata.fda.gov). […]

Battling Sanfilippo Syndrome Type A – Reed’s Story

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In January of 2011, we learned that our then three and a half year old son Reed has Sanfilippo Syndrome Type A, a rare and fatal neurodegenerative disease. The disease is very rare, affecting about 1 in 70,000 births. To put that in perspective, according […]