Ultragenyx Announces Data Demonstrating Treatment With UX111
Portfolio Pulse from Benzinga Newsdesk
Ultragenyx Pharmaceutical Inc. announced positive data from its Transpher A study on UX111 (ABO-102) AAV gene therapy for treating Sanfilippo syndrome type A (MPS IIIA). The study showed rapid and sustained decreases in heparan sulfate levels in patients, correlating with improved cognitive development. Results will be presented at the WORLDSymposium 2024.

February 06, 2024 | 1:16 pm
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POSITIVE IMPACT
Ultragenyx's positive study results for UX111 in treating Sanfilippo syndrome type A could significantly boost investor confidence and potentially lead to increased regulatory support and market opportunities.
The announcement of positive data from the Transpher A study on UX111 for treating Sanfilippo syndrome type A by Ultragenyx (RARE) is likely to be viewed positively by investors and could lead to increased regulatory support. This, in turn, may open up significant market opportunities for the company, positively impacting its stock price in the short term.
CONFIDENCE 80
IMPORTANCE 85
RELEVANCE 90