Ultragenyx Pharmaceutical Announces FDA Acceptance of UX016 IND [Yahoo! Finance]
Ultragenyx Pharmaceutical Inc. (RARE)
Last ultragenyx pharmaceutical inc. earnings: 2/13 04:01 pm
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US:NASDAQ Investor Relations:
ir.ultragenyx.com/investor-relations
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Source: Yahoo! Finance
drug (IND) application for UX016, an investigational small-molecule prodrug of sialic acid, which is being developed as a substrate replacement therapy for GNE myopathy (GNEM). GNEM, also known as hereditary inclusion body myopathy or Nonaka myopathy, is a rare inherited neuromuscular disorder caused by reduced production of sialic acid. This deficiency leads to progressive muscle wasting, resulting in severe disability and loss of mobility. Currently, there are no approved treatments for this disease in the United States. Year to date, RARE's shares have lost 16.1% against the industry's 0.3% growth. Zacks Investment Research Image Source: Zacks Investment Research Clinical Development Plan The program is being externally funded by a patient organization through early clinical development, with the first-in-human phase I/II study expected to be initiated in the second half of 2026 in the United States. Study Design & Endpoints The study will enroll approximately 24 ad
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RARE
News
- Ultragenyx (RARE) Shares Positive Results From DTX301 Phase 3 Study [Yahoo! Finance]Yahoo! Finance
- Ultragenyx Pharmaceutical Inc. Notice of April 6, 2026 Application Deadline for Class Action Lawsuit - Contact Lewis Kahn, Esq. at Kahn Swick & Foti, LLC, Before Application DeadlineGlobeNewswire
- INVESTOR ALERT: Pomerantz Law Firm Reminds Investors with Losses on their Investment in Ultragenyx Pharmaceutical Inc. of Class Action Lawsuit and Upcoming Deadlines – RAREGlobeNewswire
- RARE Shareholder Alert: Ultragenyx Pharmaceutical Inc. Securities Class Action Lawsuit Investors With Losses May Join -- The Gross Law FirmPR Newswire
- Portnoy Law Firm Announces Class Action on Behalf of Ultragenyx Pharmaceutical, Inc. InvestorsGlobeNewswire
RARE
Earnings
- 2/12/26 - Miss
RARE
Sec Filings
- 3/30/26 - Form 8-K
- 3/27/26 - Form ARS
- 3/27/26 - Form DEFA14A
- RARE's page on the SEC website