Why the WHO Global Action Plan matters for Rare disease communities. Jane Cooper, senior vice president and region head, Europe, Middle East and Africa, and Eduardo Thompson, senior vice president and region head, Latin America, share Ultragenyx's contribution to the development of a WHO Global Action Plan for Rare Diseases. Jane Cooper and Eduardo Thompson Across Europe, Middle East, Africa and Latin America, Ultragenyx Pharmaceutical Inc. meet people living with rare diseases and their families whose journeys began long before they received a diagnosis. From Chile to UAE, parents of children impacted by rare genetic diseases often spend years searching for answers. Patients travel across borders to find specialist care. Healthcare professionals struggle to find answers for unknown conditions. That is why Ultragenyx Pharmaceutical Inc. believe last year's World Health Assembly Resolution for a Global Action Plan for Rare Diseases is an important moment for the rare disease community. As Ultragenyx, Ultragenyx Pharmaceutical Inc. is proud to work with partners in Europe and Latin American on the development of patient-voice led draft Action Plans for its respective World Health Organization regions. For the first time, there is an opportunity to create a shared framework for improving the lives of more than 300 million people living with a rare disease worldwide. Together as patients and caregiver communities, clinicians, researchers, policymakers and companies Ultragenyx Pharmaceutical Inc. is coalescing around a common goal. At Ultragenyx, its mission is to transform the lives of people living with rare and ultra-rare diseases. Over the past decade, Ultragenyx Pharmaceutical Inc. has brought new treatments to people living with rare disease who did not have treatments before. Ultragenyx Pharmaceutical Inc. has also helped advance new approaches to improve rare disease drug development, initiating 12 new evaluation tools based on patient feedback. Ultragenyx Pharmaceutical Inc. do not shy away from adaptive trial designs and seamless Phase 1/2/3 trials to accelerate bringing new treatments to patients. Ultragenyx Pharmaceutical Inc. look at the totality of the disease, encouraging regulators to consider multiple endpoints to address patient variability and breadth of disease in small patient populations. Ultragenyx Pharmaceutical Inc. believe that scientific innovation is only as meaningful as the lives it changes. Healthcare systems must be able to find patients, connect them with the right expertise and support the full patient journey through access to trained healthcare professionals, appropriate institutions, coordinated services and treatment choice where available. This is the north star of its participation in multi-stakeholder discussions across Europe and Latin America around the future WHO Global Action Plan. These are the ambitions Ultragenyx Pharmaceutical Inc. want to hold ourselves to account for. Are patients getting accurate diagnoses as soon as possible? For many people living with a rare disease, the greatest challenge is obtaining an accurate diagnosis. Whether speaking with families affected by Sanfilippo syndrome, Homozygous familial hypercholesterolaemia (HoFH), or other rare genetic conditions, Ultragenyx Pharmaceutical Inc. regularly hear stories of years spent searching for answers. Earlier diagnosis can open the door to specialist care, support services, research opportunities and, where available, treatment. Once a treatment is available, early diagnosis - including at birth - of certain diseases is critical to realise the full benefit a treatment can bring. Reducing the diagnostic odyssey has to be one of the defining ambitions of the Global Action Plan. Have Ultragenyx Pharmaceutical Inc. strengthened the rare disease ecosystems? Rare and ultra-rare diseases present unique challenges because expertise, data and patients are often dispersed across countries and continents. In Europe, Ultragenyx Pharmaceutical Inc. has seen how investment in the European Reference Network for shared knowledge, cross-border research partnerships and care delivery can generate greater equity. The European Reference Network created the largest formal rare disease expertise network in Europe by connecting 375 hospitals and more than 1600 specialised centres in 28 countries to enable access to highly specialised clinical knowledge regardless of where a patient lives. The Global Action Plan offers an opportunity to build on this global success story and globalise the impact of shared expertise, patient registries and research networks in helping patients access knowledge and support that may not exist locally. Is innovation reaching patients? As a company focused exclusively on rare and ultra-rare diseases, Ultragenyx Pharmaceutical Inc. understand both the promise and the complexity of developing therapies for very small patient populations. Ultragenyx Pharmaceutical Inc. believe the Global Action Plan can play an important role in harmonising the structures, processes and agencies that underpin its research journey, approvals timelines and patient access. Ultragenyx Pharmaceutical Inc. believe the Global Action Plan can create collaboration within and between regions and countries on evaluating the scientific and health economic merits of rare and ultra-rare treatments with no other treatment options. It can showcase best practice in funding treatments and creating pathways that countries can choose to replicate as part of their national rare disease plans. Most importantly, a strong ambition of support for innovation will unlock more research and investment into those rare diseases with no current treatment options. While healthcare systems differ significantly across the world and even within its regions in Europe, Middle East, Africa, Central and Latin America, the aspirations of rare disease families are strikingly similar. They want answers. They want access to expertise. They want opportunities to participate in research. And they want hope for the future. That is what One Rare Nation means to Ultragenyx Pharmaceutical Inc.. And that is why this moment matters. Find more about the WHO Global Action Plan for Rare Diseases here. Jane Cooper Senior vice president and region head, Europe, Middle East and Africa. Eduardo Thompson Senior vice president and region head, Latin America

Ultragenyx Pharmaceutical Inc. $RARE shares sold by Hsbc Holdings PLC. July 16, 2026 Key points. * Hsbc Holdings PLC cut its Ultragenyx Pharmaceutical stake by 61.4% in the fourth quarter, leaving it with 29,679 shares worth about $663,000. * Ultragenyx reported a wider-than-expected quarterly loss of ($1.84) per share on revenue of $136 million, missing analyst estimates on both earnings and sales. * Despite mixed fundamentals, Wall Street remains cautiously positive, with a Moderate Buy consensus and an average price target of $58.41, while several insiders also sold shares recently. * MarketBeat previews top five stocks to own in August. Hsbc Holdings PLC lessened its stake in Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE - Free Report) by 61.4% in the fourth quarter, according to its most recent filing with the Securities & Exchange Commission. The institutional investor owned 29,679 shares of the biopharmaceutical company's stock after selling 47,206 shares during the period. Hsbc Holdings PLC's holdings in Ultragenyx Pharmaceutical were worth $663,000 at the end of the most recent quarter. Several other large investors have also recently bought and sold shares of the stock. UBS AM A Distinct Business Unit of UBS Asset Management Americas LLC boosted its holdings in shares of Ultragenyx Pharmaceutical by 14.8% in the first quarter. UBS AM A Distinct Business Unit of UBS Asset Management Americas LLC now owns 232,360 shares of the biopharmaceutical company's stock valued at $8,414,000 after acquiring an additional 29,984 shares in the last quarter. Creative Planning bought a new stake in Ultragenyx Pharmaceutical during the second quarter worth approximately $454,000. American Century Companies Inc. acquired a new stake in Ultragenyx Pharmaceutical in the second quarter worth approximately $366,000. M&T Bank Corp lifted its position in Ultragenyx Pharmaceutical by 19.7% in the second quarter. M&T Bank Corp now owns 7,232 shares of the biopharmaceutical company's stock worth $263,000 after purchasing an additional 1,192 shares during the period. Finally, Amundi boosted its stake in Ultragenyx Pharmaceutical by 1,560.4% during the 2nd quarter. Amundi now owns 99,161 shares of the biopharmaceutical company's stock valued at $3,913,000 after purchasing an additional 93,189 shares in the last quarter. Institutional investors and hedge funds own 97.67% of the company's stock. Ultragenyx Pharmaceutical stock down 1.0%. Shares of NASDAQ:RARE opened at $31.71 on Thursday. The company's fifty day moving average is $27.14 and its two-hundred day moving average is $24.45. The company has a market cap of $3.12 billion, a P/E ratio of -5.19 and a beta of 0.31. Ultragenyx Pharmaceutical Inc. has a 12-month low of $18.29 and a 12-month high of $39.89. Ultragenyx Pharmaceutical (NASDAQ:RARE - Get Free Report) last issued its quarterly earnings results on Tuesday, May 5th. The biopharmaceutical company reported ($1.84) earnings per share for the quarter, missing analysts' consensus estimates of ($1.49) by ($0.35). The business had revenue of $136.00 million for the quarter, compared to analysts' expectations of $158.19 million. Ultragenyx Pharmaceutical had a negative net margin of 91.03% and a negative return on equity of 1,024.42%. The business's quarterly revenue was down 2.2% compared to the same quarter last year. During the same quarter in the prior year, the firm posted ($1.57) EPS. Equities research analysts anticipate that Ultragenyx Pharmaceutical Inc. will post -4.53 earnings per share for the current year. Insiders place their bets. In other news, EVP Karah Herdman Parschauer sold 1,899 shares of the stock in a transaction that occurred on Monday, June 15th. The shares were sold at an average price of $24.62, for a total value of $46,753.38. Following the completion of the sale, the executive vice president directly owned 94,462 shares of the company's stock, valued at $2,325,654.44. This represents a 1.97% decrease in their ownership of the stock. The transaction was disclosed in a document filed with the Securities & Exchange Commission, which can be accessed through this link. Also, Director Shehnaaz Suliman sold 5,740 shares of the firm's stock in a transaction that occurred on Monday, May 18th. The stock was sold at an average price of $25.12, for a total transaction of $144,188.80. Following the sale, the director owned 27,951 shares of the company's stock, valued at approximately $702,129.12. This represents a 17.04% decrease in their ownership of the stock. The SEC filing for this sale provides additional information. Insiders have sold 19,319 shares of company stock worth $476,837 over the last quarter. Company insiders own 5.20% of the company's stock. Analysts set new price targets. Several brokerages have recently issued reports on RARE. Wedbush lowered their price target on Ultragenyx Pharmaceutical from $27.00 to $26.00 and set a "neutral" rating on the stock in a research report on Wednesday, May 6th. Guggenheim cut their price objective on Ultragenyx Pharmaceutical from $52.00 to $43.00 and set a "buy" rating for the company in a report on Friday, May 8th. Weiss Ratings raised Ultragenyx Pharmaceutical from a "sell (e+)" rating to a "sell (d-)" rating in a research note on Thursday, July 9th. Truist Financial raised Ultragenyx Pharmaceutical to a "strong-buy" rating in a report on Wednesday, March 25th. Finally, Wall Street Zen upgraded shares of Ultragenyx Pharmaceutical from a "strong sell" rating to a "sell" rating in a research report on Saturday, June 6th. One equities research analyst has rated the stock with a Strong Buy rating, fourteen have issued a Buy rating, two have assigned a Hold rating and one has given a Sell rating to the company. According to data from MarketBeat.com, the company has a consensus rating of "Moderate Buy" and an average target price of $58.41. Discover more American Consumer News Cryptocurrency market updates About Ultragenyx Pharmaceutical. Ultragenyx Pharmaceutical Inc is a biopharmaceutical company focused on developing and commercializing therapies for rare and ultra-rare genetic disorders. Since its founding in 2010 and headquarters in Novato, California, the company has built expertise in protein replacement therapies, small molecules and gene therapy approaches to address high-unmet medical needs. Ultragenyx applies a precision medicine model, leveraging both in-house research and strategic collaborations to advance its product pipeline from discovery through regulatory approval. The company's commercial portfolio includes Crysvita (burosumab-tmyl) for X-linked hypophosphatemia, Mepsevii (vestronidase alfa-vjbk) for mucopolysaccharidosis VII and Dojolvi (triheptanoin) for long-chain fatty acid oxidation disorders. This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to [email protected]. Before you consider Ultragenyx Pharmaceutical, you'll want to hear this. MarketBeat keeps track of Wall Street's top-rated and best performing research analysts and the stocks they recommend to their clients on a daily basis. MarketBeat has identified the five stocks that top analysts are quietly whispering to their clients to buy now before the broader market catches on... and Ultragenyx Pharmaceutical wasn't on the list. 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Spruce Biosciences appoints Agnieszka Jurecka, M.D., Ph.D., MPH, as Vice President, Medical Affairs. Dr. Jurecka Brings Deep Enzyme Replacement Therapy and Rare Disease Experience to Lead Medical Affairs and Scientific Engagement Ahead of Potential U.S. FDA Approval of Tralesinidase Alfa Enzyme Replacement Therapy (TA-ERT) for the Treatment of Sanfilippo Syndrome Type B (MPS IIIB) SOUTH SAN FRANCISCO, Calif.-(BUSINESS WIRE)-Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need, today announced the appointment of Agnieszka "AJ" Jurecka, M.D., Ph.D., MPH, as Vice President, Medical Affairs. "We are delighted to welcome AJ to the Spruce executive team. She is a rare combination of practicing physician, scientist, and biopharmaceutical leader, with directly relevant enzyme replacement therapy and lysosomal storage disorder experience," said Kirk Ways, M.D., Ph.D., Chief Medical Officer of Spruce Biosciences. "With a potential U.S. approval anticipated next year, AJ gives us the medical affairs and scientific engagement leadership to establish disease awareness, build durable KOL relationships, and execute launch readiness for TA-ERT." Dr. Jurecka brings more than 20 years of clinical medicine and biopharmaceutical industry experience focused on rare and ultra-rare genetic diseases, including lysosomal storage disorders, inborn errors of metabolism, and neurogenetic conditions, with deep expertise spanning enzyme replacement therapies, small molecules, and gene therapy. She has led global clinical development and medical affairs programs from pre-IND through late-stage and Phase IV, and has supported regulatory interactions with the FDA, EMA, and other global health authorities. Most recently, Dr. Jurecka has served as Chief Medical Officer to a seed-stage gene therapy company developing CNS-directed therapies for lysosomal gene defects, including CLN1 and CLN2. Previously, she served as Vice President, Clinical Development at CoA Therapeutics (a BridgeBio company), where she led global clinical development strategy for rare neurodegenerative and metabolic disease programs from pre-IND through Phase 1 and into pivotal trial planning. Earlier, Dr. Jurecka served as Senior Medical Director, Global Clinical Development at Ultragenyx Pharmaceutical, supporting development and medical affairs for the mucopolysaccharidosis type VII (MPS VII) enzyme replacement therapy program and long-chain fatty acid oxidation disorders. Earlier in her career, she held medical affairs leadership roles at Aegerion Pharmaceuticals, Shire Pharmaceuticals (MPS II enzyme replacement therapy programs, including intrathecal ERT), Synageva BioPharma (lysosomal acid lipase deficiency ERT), and BioMarin Europe (Naglazyme for MPS VI). She is the author of more than 60 peer-reviewed publications in rare disease and lysosomal storage disorders. Dr. Jurecka earned her M.D., magna cum laude, from the Medical University of Silesia, her Ph.D. in Medicine from the Children's Memorial Health Institute in Warsaw, Poland, and MPH from Harvard T.H. Chan School of Public Health. "Sanfilippo Syndrome Type B is a devastating disease, and TA-ERT has the potential to be the first disease-modifying therapy for the children and families affected by it," said Dr. Jurecka. "Having spent much of my career in rare disease drug development and medical affairs across enzyme replacement therapies and lysosomal storage disorders, I am honored to join Spruce at this defining moment and to help build the medical and scientific foundation that will support TA-ERT through approval and launch." Inducement Award In connection with Dr. Jurecka's employment with Spruce, on June 22, 2026, Dr. Jurecka was granted restricted stock units (RSUs) for 3,800 shares of Spruce's common stock. The Compensation Committee of the Board of Directors approved the awards as inducements material to Dr. Jurecka's entering into employment with the company in accordance with Nasdaq Listing Rule 5635(c)(4). The RSUs will vest over four years, with 25% of the underlying shares vesting on each anniversary of June 15, 2026, subject to Dr. Jurecka's continued service relationship with Spruce Biosciences through the applicable vesting date. The award will be subject to the terms and conditions of the 2026 Inducement Plan and the terms and conditions of the applicable award agreement covering the grant. About Spruce Biosciences Spruce Biosciences is a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need. Spruce's lead product candidate, tralesinidase alfa enzyme replacement therapy (TA-ERT), is in late-stage development for the treatment of mucopolysaccharidoses type IIIB (MPS IIIB), or Sanfilippo Syndrome Type B, a devastating pediatric neurodegenerative disorder for which there are no FDA-approved therapies. TA-ERT has received Breakthrough Therapy Designation, Rare Pediatric Disease Designation, Fast Track Designation and Orphan Drug Designation from the FDA, as well as Orphan Drug Designation in the European Union. To learn more, visit www.sprucebio.com and follow us on X, LinkedIn, Facebook and YouTube. Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the impact of new management hires, the fulfillment of Spruce's strategic business objectives, potential regulatory approval, commercial launch of TA-ERT, and TA-ERT's potential to be the first disease-modifying treatment option for MPS IIIB. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "plan," "will," "believe," "could," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Spruce's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Spruce's business in general, the impact of geopolitical and macroeconomic events, and the other risks described in Spruce's filings with the U.S. Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. Spruce undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. More News From Spruce Biosciences, Inc. SOUTH SAN FRANCISCO, Calif.-( BUSINESS WIRE )-Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need, today announced the appointments of Eric Jordan as Vice President, Marketing and Tina Gullotta, CPA, as Vice President, Finance and Corporate Controller."We are delighted to welcome both Eric and Tina, two accomplished leaders with deep biotech experie... SOUTH SAN FRANCISCO, Calif.-( BUSINESS WIRE )-Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need, today announced that data demonstrating the long-term benefit of tralesinidase alfa enzyme replacement therapy (TA-ERT) in patients with Sanfilippo Syndrome Type B (MPS IIIB) were presented at the 18th International MPS & Related Lysosomal Disease... SOUTH SAN FRANCISCO, Calif.-( BUSINESS WIRE )-Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need, today announced that data on the long-term administration of its tralesinidase alfa enzyme replacement therapy (TA-ERT) for the treatment of Sanfilippo Syndrome Type B (MPS IIIB) will be presented at the 18th International MPS & Related Lysosomal... Spruce Biosciences, Inc. NASDAQ:SPRB Release Versions