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Tag: Sanfilippo Syndrome Type A

A total of 2 posts are filed under Sanfilippo Syndrome Type A
lysogene
Lysogene Recieves US Orphan Designation for its Lead Intracerebral Gene Therapy Product SAF-301
LYSOGENE announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its lead gene therapy… Continue Reading
Reed_MPS_SanFilippo
Battling Sanfilippo Syndrome Type A – Reed’s Story
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… Continue Reading

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