For some families, this is a daily reality due to rare genetic disorders. One such condition is Sanfilippo syndrome, which creates significant hurdles for affected children and their families.

Ultragenyx Pharmaceutical RARE reported new findings from an ongoing, pivotal study of its investigational candidate, UX111 (ABO-102) AAV gene therapy, for Sanfilippo syndrome type A (MPS IIIA).

(RARE), a biopharmaceutical company, on Wednesday reported encouraging data from its ongoing clinical program of UX111, a gene therapy for Sanfilippo Syndrome Type A or MPS IIIA, a rare ...

Jeffrey Sanfilippo; Chairman of the Board, Chief Executive Officer; John B Sanfilippo & Son Inc Frank Pellegrino; Chief Financial Officer, Executive Vice President - Finance and Administration ...

Tourette Syndrome is “a disorder that involves repetitive movements or unwanted sounds (tics) that can't be easily controlled,” according to the Mayo Clinic. In Baylen’s case, she often ...

An Orange couple who lost their son to a rare genetic condition are determined to continue raising awareness to improve the lives of diagnosed children and their families.

I would like to say that I don’t tic 24/7, but it consumes a lot of my day and a lot of my time." Tourette syndrome (TS) is “a disorder that involves repetitive movements or unwanted sounds ...

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