Ultragenyx Announces Positive Longer-term Data from Phase 3 Study of DTX401 AAV Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa)
Ultragenyx Pharmaceutical Inc. (RARE)
Last ultragenyx pharmaceutical inc. earnings: 2/13 04:01 pm
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Source: GlobeNewswire
At Week 96 participants experienced even greater reductions in daily cornstarch intake while maintaining low levels of hypoglycemia, improved levels of euglycemia and improved fasting tolerance Phase 3 results are further supported by early data from open-label Japanese cohort (n=3) where all participants were able to eliminate daily cornstarch while maintaining or improving glycemic control DTX401 was well tolerated with an acceptable safety profile NOVATO, Calif., Sept. 08, 2025 (GLOBE NEWSWIRE) -- Today, Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced positive longer-term results from its Phase 3 study of DTX401 AAV gene therapy for the treatment of glycogen storage disease type Ia (GSDIa). Previously reported 48 Week data show that patients treated with DTX401 (n=20) experienced statistically significant and clinically meaningful reductions in daily cornstarch intake compared to placebo, while maintaining glycemic control. At Week 96, even greater reductions in
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News
- Ultragenyx (RARE): Assessing Valuation as Revenue Growth and Clinical Progress Fuel Renewed Investor Interest [Yahoo! Finance]Yahoo! Finance
- Ultragenyx to Participate in Investor Conferences in DecemberGlobeNewswire
- Ultragenyx Pharmaceutical (NASDAQ:RARE) had its price target lowered by analysts at Barclays PLC from $81.00 to $50.00. They now have an "overweight" rating on the stock.MarketBeat
- Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) [Yahoo! Finance]Yahoo! Finance
- Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)GlobeNewswire
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