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Congratulations, Ultragenyx! The FDA recently agreed with Ultragenyx that cerebral spinal fluid heparan sulfate can be a surrogate endpoint for Sanfilippo Syndrome. This can now be used in Ultragenyx’s biologics license application as the company seeks accelerated approval for its gene therapy to treat the rare disease. It’s been a privilege to partner with Ultragenyx by designing the facility that will produce these treatments. Here’s to helping their goal of treating rare and ultrarare diseases in our world! Learn more here: https://lnkd.in/eVe84geG #LifeSciences #GeneTherapy #RareDisease

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