St. Jude Children's Research Hospital, Inc.

Advisors to the FDA had difficulty dissecting a small but concerning set of safety data from Moderna’s pediatric RSV vaccine data, largely concluding that other trials should continue to better inform immune responses in young kids. The FDA has flagged concerns based on severe cases of RSV seen in vaccinated children in Moderna’s Phase 1 study of two experimental vaccines, potentially due to vaccine-associated enhanced respiratory disease. The cases harken back to clinical studies in the 1960s, when two toddlers died in a vaccine trial, stalling pediatric RSV work for decades. Then, as now, severe RSV cases occurred in children who had never previously been infected with the virus. “We’re confronted by a very complicated situation,” said Arnold Monto, a professor emeritus of epidemiology at the University of Michigan. He suggested that the agency should consider safety for the individual platforms, but not the class as a whole. The Vaccines and Related Biological Products Advisory Committee wasn’t asked to vote Thursday, but rather to provide feedback on whether available data suggest a general safety risk to infants and toddlers or whether it’s unique to specific vaccine platforms. In July, the FDA put a clinical hold on Moderna’s study after severe RSV cases arose in five patients who received one of two vaccine candidates. By early August, the agency put a partial hold on other pediatric studies involving vaccine candidates that were not based on a live-attenuated vaccine. A sixth patient reported a severe RSV case months after completing vaccination and after the FDA placed the holds. The FDA didn’t respond to a question about how many studies were placed on a partial hold, but the agency outlined in briefing documents that 11 candidates that did not involve a live-attenuated vaccine candidate had pediatric studies. The holds weren’t disclosed by either the agency or the companies until the briefing materials were released ahead of the meeting this week. The FDA hasn’t yet identified a similar safety signal from other vaccine candidates, despite the partial hold. Octavio Ramilo, chair of infectious diseases at St. Jude Children’s Research Hospital, was brought on by the data safety monitoring board of Moderna’s trial to better understand the data. He acknowledged that the understanding of how to make a vaccine and leverage preclinical animal models was incomplete. “So I think there [are] a lot of gaps in our knowledge,” he said. ‘The monitoring here worked’ Moderna reported no cases of severe RSV hospitalization through March 2024 in the portion of the trial testing infants 8 to 23 months, and the company then moved to a younger cohort of patients who were 5 to 7 months old. Since the trial was halted, Moderna has also reported a case in the older population, which arose in August. Four of five severe cases included hospitalization, and all but one patient has gotten better. The most severe patient was hospitalized for 16 days, including 10 in the ICU, and that patient still had arterial hypertension. Moderna announced in September that it was not advancing the pediatric vaccine, but did not disclose at the time that it was under clinical hold. A spokesperson for the company declined to say why the hold wasn’t disclosed then when reached for comment earlier this week. The advisors seemed to largely endorse the regulatory safeguards in place, despite Moderna reporting encouraging preclinical safety data in cotton mice ahead of the clinical trials. Advisor Adam Berger said, “the monitoring here worked.” “The safety signal was identified. The sponsor halted the study. This is exactly the type of safeguards you want to see happen,” he said. Sarah Meyer, chief medical officer at the Immunization Services Division within the CDC, underscored that it’s difficult to assess trial safeguards without further understanding the mechanism of action that may have spurred the severe cases. Blue Lake Biotechnology, which was included in the subsequent partial hold, presented to the committee that it had not “detected any safety signals” of severe disease associated with its vaccine, according to Chief Medical Officer Henry Radziewicz. The company similarly had not publicly disclosed that it was on a partial hold before Thursday’s meeting. Sanofi also presented on its live-attenuated vaccine, currently in a Phase 3 study, saying that it had not seen any concerning safety signals. It’s not included in the FDA’s partial holds and committee members seemed to be assuaged by the fact that it has been given to many more patients in trials. “I’m not worried about the live-attenuated [vaccines] because of the long history of safety there,” said Tracy Ruckwardt, chief of the respiratory viruses core at the NIH’s Vaccine Research Center. Editor’s note: This story was updated to correct that Moderna’s trial was a Phase 1 study, not a Phase 1/2 study.

– Data from the pivotal Phase 2b ReNeu trial demonstrate that patients with NF1-PN achieved deep responses and improvements in health-related quality of life over the course of mirdametinib treatment – – Encouraging results from Phase 1/2 study of mirdametinib in pediatric patients with low-grade glioma also to be presented in oral session at SNO – STAMFORD, Conn., Nov. 11, 2024 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, today announced that three abstracts from the pivotal Phase 2b ReNeu trial of mirdametinib, an investigational MEK inhibitor, in adults and children with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN) will be presented in oral and poster sessions at the 29th Annual Meeting & Education Day of the Society for Neuro-Oncology (SNO), being held November 21-24, 2024. ReNeu (NCT03962543) is a multicenter, single-arm trial and the largest study conducted to date in patients with NF1-PN. As previously reported, data from the ReNeu trial demonstrated deep and sustained reductions in tumor volume as well as improvement in pain and health-related quality of life (HRQoL) in both the adult and pediatric cohorts. New data being presented at SNO show that the deep responses in tumor volume reduction were achieved regardless of baseline characteristics, and suggest a trend between deep response and both earlier achievement of a first confirmed response and longer treatment duration. In addition, the improvements in HRQoL were clinically meaningful, early, and sustained over the course of mirdametinib treatment. Data from the Phase 1/2 trial of mirdametinib in pediatric and young adult patients with low-grade gliomas (LGG) will also be presented in an oral presentation at SNO and suggest that mirdametinib is well-tolerated and has promising clinical activity in this patient population, including a 63% objective response rate in patients with measurable tumors and a median time to response of 5.4 months. “We are very pleased that new data analyses from our ReNeu trial continue to support the potentially differentiated profile of mirdametinib for patients with NF1-PN, including deeper responses in target tumors for those who were on treatment for a longer duration, and meaningful improvements across quality-of-life measures,” said Jim Cassidy, M.D., Ph.D., Chief Medical Officer of SpringWorks Therapeutics. “We are also encouraged by the data in children and young adults with LGG treated with mirdametinib and look forward to the phase 2 portion of the trial to further evaluate the efficacy and safety of mirdametinib in this patient population.” A New Drug Application (NDA) for mirdametinib in adults and children with NF1-PN was granted Priority Review designation by the U.S. Food and Drug Administration (FDA), with a Prescription Drug User Fee Act action date of February 28, 2025. In addition, the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for mirdametinib for the treatment of adult and pediatric patients with NF1-PN. Oral and Poster Presentations at 2024 SNO Annual Meeting Pivotal, phase 2b ReNeu trial of mirdametinib in children and adults with neurofibromatosis type-1 associated plexiform neurofibroma (NF1-PN): A spotlight on patients achieving deep responsePoster PresentationAbstract #: CTNI-21Date and Time: November 22, 7:30-9:30 p.m. CST (8:30-10:30 p.m. EST) As previously reported at the 2024 American Society of Clinical Oncology Annual Meeting, the Phase 2b ReNeu trial met its primary endpoint of confirmed objective response rate, as assessed by blinded independent central review, in both adults and children. Tumor volume reductions were deep and durable over the course of the study. The data being presented at SNO demonstrate that the deep responses in target tumors were achieved regardless of different baseline characteristics and also show that patients with a deep response had longer treatment duration with mirdametinib. The SNO data include: Of the 41% (24/58) of adults and 52% (29/56) of children who experienced a confirmed objective response during the 24-cycle treatment phase (approximately 22 months), 62% (15/24) of adults and 52% (15/29) of children achieved deep response (defined as >50% best reduction from baseline in target PN volume).Of those with a deep response in the total cohort, 35% of adults (6/17) and 72% of children (13/18) were investigator-defined as having progressing PN at baseline.Patients achieved deep response regardless of age, sex, target PN volume, tumor location, or progression status at baseline.The median time to best percent change from baseline in PN volume for patients achieving deep response was 25 months for adults and 22 months for children. For patients with ≥20% to ≤50% PN volume reduction, the median time to best percent change from baseline was 15 months for adults and 15 months for children. “It is very encouraging to see such deep tumor volume reductions across subgroups of patients, and the trend we observed between deep response and longer treatment duration suggests that patients can benefit from prolonged therapy with mirdametinib,” said Timothy R. Gershon, M.D., Ph.D., professor in the Department of Pediatrics at Emory University School of Medicine, Director of the Children’s Center for Neurosciences Research at Emory University, and ReNeu trial investigator. “The collective findings from the ReNeu trial support the potential for mirdametinib to be a much-needed therapy for patients with this debilitating disease.” Health-related quality-of-life (HRQoL) in adults and children with neurofibromatosis type-1 associated plexiform neurofibroma (NF1-PN) treated with mirdametinib: Pivotal, phase 2b ReNeu trialOral Presentation Abstract #: QOL-08Date and Time: November 24, 10:25-10:35 a.m. CST (11:25-11:35 a.m. EST) In the ReNeu trial, change in HRQoL in adults and children was assessed by the Pediatric QoL Inventory (Peds QL) Total Score; change from baseline at Cycle 13 was a prespecified secondary endpoint. Results showed clinically meaningful, early, and sustained benefits in HRQoL, including: Improvement (least-squares mean, LSM [SE] change) from baseline at Cycle 13 was 3.9 (1.6; P=.018; n=34) for adults, 4.0 (2.4; P=.096; n=38) for children by patient-report, and 5.6 (1.9; p=0.005; n=43) by parent proxy-report.Improvements for adults and children by parent proxy-report were observed early (at Cycle 5 and Cycle 3, respectively) and sustained at most time points through Cycle 13.Clinically meaningful improvement from baseline at Cycle 13 was achieved by 37% (10/27) of adults, 45% (13/29) of children by patient-report, and 47% (15/32) of children by parent proxy-report (among patients who could have achieved a clinically meaningful change from baseline). “Patients with NF1-PN experience pain and other symptoms that negatively impact their functioning and quality of life,” said Rene Y. McNall-Knapp, M.D., a pediatric hematologist-oncologist at the Jimmy Everest Center at Oklahoma Children’s Hospital OU Health and ReNeu study investigator. “In the ReNeu trial, both adults and children experienced early and sustained improvements in health-related quality of life over the course of treatment with mirdametinib, which is an important outcome of treatment for those living with this devastating disease.” Addressing skin adverse events (AEs) during mirdametinib treatment in patients with neurofibromatosis type-1 associated plexiform neurofibroma (NF1-PN): Guidance from a multidisciplinary group of experts on the management of MEK inhibitor-associated skin AEsPoster PresentationAbstract #: CTNI-20Date and Time: November 22, 7:30-9:30 p.m. CST (8:30-10:30 p.m. EST) Skin adverse events (AEs) are commonly seen with MEK inhibitors as a class and were common in the ReNeu trial. To prevent and manage skin AEs, a multidisciplinary team retrospectively reviewed skincare practices at one high-enrolling ReNeu trial site and provided a series of recommendations to healthcare professionals to support treatment adherence. Results from the Phase 1 and Phase 1 expansion cohorts of SJ901: A Phase 1/2 trial of single-agent mirdametinib (PD-0325901) in children, adolescents, and young adults with low-grade gliomaOral PresentationAbstract #: CTNI-70Date and Time: November 22, 11:50-11:55 a.m. CST (12:50-12:55 p.m. EST) Data from the Phase 1 and Phase 1 expansion cohorts of an ongoing Phase 1/2 trial (NCT04923126) evaluating mirdametinib in patients ages 2 to 24 with pediatric and young adult low-grade gliomas (LGG) suggest that mirdametinib, which has high blood brain barrier penetration, is well tolerated and has promising clinical activity in patients with recurrent/progressive LGG across a variety of MAPK pathway aberrations. Results demonstrated: Of the 23 patients enrolled in the trial, 17 (74%) completed or remain on-therapy; 4 (17%) stopped for progression, and two discontinued for toxicities.Twelve (63%) of the 19 patients with measurable tumors achieved an objective response (one major, six partial, and five minor responses).The median time to an objective response was 5.4 months (range: 1.7 to 7.3).Mirdametinib was well-tolerated in the Phase 1 portion of the trial. This trial is being conducted pursuant to a research agreement that SpringWorks entered into with St. Jude Children’s Research Hospital. These data were previously presented at the 21st International Symposium on Pediatric Neuro-Oncology (ISPNO 2024). The Phase 2 portion of the trial is ongoing and recruiting patients. About the ReNeu Trial ReNeu (NCT03962543) is an ongoing, multi-center, open-label, single arm, Phase 2b trial evaluating the efficacy, safety, and tolerability of mirdametinib in patients ≥2 years of age with an inoperable NF1-associated PN causing significant morbidity. The study enrolled 114 patients to receive mirdametinib at a dose of 2 mg/m2 twice daily (maximum dose of 4 mg twice daily) without regard to food. Mirdametinib was administered orally in a 3-week on, 1-week off dosing schedule as either a capsule or dispersible tablet. The primary endpoint is confirmed objective response rate defined as the proportion of patients with a ≥ 20% reduction in target tumor volume on consecutive scans during the 24-cycle treatment phase, as measured by MRI and assessed by blinded independent central review. Secondary endpoints include safety and tolerability, duration of response, and changes in patient reported outcomes from baseline to Cycle 13. The treatment phase of the trial is complete, and results were presented at the 2024 American Society of Clinical Oncology Annual Meeting. Patients who completed the treatment phase were eligible to continue receiving treatment in the optional long-term follow up portion of the study, which is ongoing. About NF1-PN Neurofibromatosis type 1 (NF1) is a rare genetic disorder that arises from mutations in the NF1 gene, which encodes for neurofibromin, a key suppressor of the MAPK pathway.1,2 NF1 is the most common form of neurofibromatosis, with an estimated global birth incidence of approximately 1 in 2,500 individuals, and approximately 100,000 patients living with NF1 in the United States.3,4 The clinical course of NF1 is heterogeneous and manifests in a variety of symptoms across numerous organ systems, including abnormal pigmentation, skeletal deformities, tumor growth and neurological complications, such as cognitive impairment.5 Patients with NF1 have an 8 to 15-year mean reduction in their life expectancy compared to the general population.3 NF1 patients have approximately a 30-50% lifetime risk of developing plexiform neurofibromas, or PN, which are tumors that grow in an infiltrative pattern along the peripheral nerve sheath and that can cause severe disfigurement, pain and functional impairment; in rare cases, NF1-PN may be fatal.6,7 NF1-PNs are most often diagnosed in the first two decades of life.6 These tumors can be aggressive and are associated with clinically significant morbidities; typically, they grow more rapidly during childhood.8,9 Surgical removal of these tumors is challenging due to the infiltrative tumor growth pattern along nerves and can lead to permanent nerve damage and disfigurement.10 MEK inhibitors have emerged as a validated class of treatment for NF1-PN.11 About Mirdametinib Mirdametinib is a potent, oral, CNS-penetrant, allosteric small molecule MEK inhibitor in development as a monotherapy treatment for neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN) and low-grade glioma (LGG), and as a combination therapy for the treatment of several subsets of biomarker-defined metastatic solid tumors. Mirdametinib is an investigational drug for which safety and efficacy have not been established. Mirdametinib is designed to inhibit MEK1 and MEK2, which occupy pivotal positions in the MAPK pathway. The MAPK pathway is a key signaling network that regulates cell growth and survival and plays a central role in multiple cancers and rare diseases when genetically altered. The U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for mirdametinib in adults and children with NF1-PN. The NDA was granted Priority Review designation and has been given a Prescription Drug User Fee Act (PDUFA) action date of February 28, 2025. The European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for mirdametinib for the treatment of adult and pediatric patients with NF1-PN. In addition, the FDA and the European Commission previously granted Orphan Drug designation for mirdametinib for the treatment of NF1. The FDA has also granted Fast Track designation for the treatment of patients ≥ 2 years of age with NF1-PN that are progressing or causing significant morbidity and Rare Pediatric Disease designation for the treatment of NF1. About SpringWorks Therapeutics SpringWorks is a commercial-stage biopharmaceutical company applying a precision medicine approach to developing and delivering life-changing medicines for people with severe rare diseases and cancer. OGSIVEO® (nirogacestat), approved in the United States for the treatment of adult patients with progressing desmoid tumors who require systemic treatment, is the Company’s first FDA-approved therapy. SpringWorks also has a diversified targeted therapy pipeline spanning solid tumors and hematological cancers, with programs ranging from preclinical development through advanced clinical trials. In addition to its wholly owned programs, SpringWorks has also entered into multiple collaborations with innovators in industry and academia to unlock the full potential for its portfolio and create more solutions for patients in need. For more information, visit www.springworkstx.com and follow @SpringWorksTx on X (formerly Twitter), LinkedIn, and YouTube. SpringWorks Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, relating to our business, operations, and financial conditions, including but not limited to current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our development and commercialization plans, our preclinical and clinical results, whether the preclinical and clinical results of the mirdametinib studies will meet the regulatory requirements for an approval by the FDA or by the EMA of mirdametinib for the treatment of pediatric and adult patients with NF1-PN, the potential for mirdametinib to become an important new treatment for patients with NF1-PN, our plans for seeking regulatory approval for and making mirdametinib available for NF1-PN patients, if approved, as well as relating to other future conditions. Words such as, but not limited to, “look forward to,” “believe,” “expect,” “anticipate,” “estimate,” “intend,” “plan,” “would,” “should” and “could,” and similar expressions or words, identify forward-looking statements. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks relating to: (i) our limited experience as a commercial company, (ii) the success and timing of our product development activities, including the initiation and completion of our clinical trials, (iii) the fact that topline or interim data from clinical studies may not be predictive of the final or more detailed results of such study or the results of other ongoing or future studies, (iv) the timing of our planned regulatory submissions and interactions, including the timing and outcome of decisions made by the FDA, EMA, and other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies, (v) whether FDA, EMA, or other regulatory authorities will require additional information or further studies, or may fail or refuse to approve or may delay approval of our product candidates, (vi) our ability to obtain regulatory approval of any of our product candidates or maintain regulatory approvals granted for our products, (vii) our plans to research, discover and develop additional product candidates, (viii) our ability to enter into collaborations for the development of new product candidates and our ability to realize the benefits expected from such collaborations, (ix) our ability to maintain adequate patent protection and successfully enforce patent claims against third parties, (x) our ability to establish manufacturing capabilities, and our and our collaboration partners’ abilities to manufacture our product candidates and scale production, and (xii) our ability to meet any specific milestones set forth herein. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements. For further information regarding the risks, uncertainties and other factors that may cause differences between SpringWorks’ expectations and actual results, you should review the “Risk Factors” in Item 1A of Part II of SpringWorks’ Quarterly Report on Form 10-Q for the quarter ended June 30, 2024, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks’ subsequent filings. Contacts: MediaMedia@Springworkstx.com InvestorsInvestors@Springworkstx.com References Yap YS, McPherson JR, Ong CK, et al. The NF1 gene revisited - from bench to bedside. Oncotarget. 2014;5(15):5873-5892. doi:10.18632/oncotarget.2194.Rasmussen S, Friedman J. NF1 Gene and neurofibromatosis 1. Am J Epidemiol. 2000;151(1):33-40. doi:10.1093/oxfordjournals.aje.a010118.CTF: Children’s Tumor Foundation. New and Improved: The way to talk about NF. Press release. May 9, 2023. Accessed February 2, 2024.Lee: Lee TJ, et al. Incidence and prevalence of neurofibromatosis type 1 and 2: a systematic review and meta-analysis. Orphanet J Rare Dis. 2023;18(1):292. doi:10.1186/s13023-023-02911-2)Weiss BD, Wolters PL, Plotkin SR, et al. NF106: A neurofibromatosis clinical trials consortium Phase II trial of the MEK inhibitor mirdametinib (PD-0325901) in adolescents and adults with NF1-related plexiform neurofibromas. J Clin Onc. 2021;JCO.20.02220.doi.org/10. 1200/JCO.20.02220.Prada CE, Rangwala FA, Martin LJ, et al. Pediatric plexiform neurofibromas: impact on morbidity and mortality in neurofibromatosis type 1. J Pediatr. 2012;160(3):461-467.Miller DT, Freedenberg D, Schorry E, et al. Health supervision for children with neurofibromatosis Type 1. Pediatrics. 2019;143(5):e20190660. doi: 10.1542/peds.2019-0660.Gross A, Singh G, Akshintala S, et al. Association of plexiform neurofibroma volume changes and development of clinical morbidities in neurofibromatosis 1. Neuro Oncol. 2018;20(12):1643-1651. doi:10.1093/neuonc/noy067.Nguyen R, Dombi E, Widemann B, et al. Growth dynamics of plexiform neurofibromas: a retrospective cohort study of 201 patients with neurofibromatosis 1. Orphanet J Rare Dis. 2012;7(1):75. doi:10.1186/1750-1172-7-75.Needle M, Cnaan A, Dattilo J, et al. Prognostic signs in the surgical management of plexiform neurofibroma: The Children’s Hospital of Philadelphia experience, 1974-1994. J Pediatr. 1997;131(5):678-682. doi:10.1016/s0022-3476(97)70092-1.Ferner R. Neurofibromatosis 1 and neurofibromatosis 2: a twenty first century perspective. Lancet Neurol. 2007;6(4):340-351. doi:10.1016/s1474-4422(07)70075-3.

近期，美国USnews《美国新闻与世界报道》发布了“2024~2025最佳儿童医院排行榜”，其中丹娜—法伯/波士顿儿童医院位列第二，连续 18 年荣登全美最佳儿童癌症项目榜单，自 2007 年排名开始以来，每年都被评为全美前三的儿童癌症中心，是世界上当之无愧最好的儿科医院之一！ 波士顿儿童医院血液学/肿瘤学部主任、哈佛医学院儿科 Leland Fikes 教授医学博士David A. Williams表示：“丹娜法伯/波士顿儿童医院的项目非常独特，因为它将世界一流癌症中心和国际知名儿童医院的专业知识结合在一起，为患有癌症和血液疾病的儿童提供专业护理。这项排名认可了我们能够为所有患者提供的个性化治疗方法。” 波士顿儿童医院介绍 丹娜—法伯/波士顿儿童医院（Boston Children's Hospital）汇集了两家国际知名的研究和教学机构，自 1947 年以来一直为儿科肿瘤和血液病患者提供全面的护理。哈佛医学院的附属医疗机构共享一支临床人员队伍，在波士顿儿童医院提供住院护理和手术，在丹娜—法伯癌症研究所提供大部分门诊护理。 波士顿儿童医院始于1869年，一家非盈利医疗机构。150 年以来，一直是多学科儿科护理和疑难杂症治疗领域的全球领先者，享誉全球的医生和护士团队每年会安排超过 650,000 次患者就诊并实施超过 27,000 例外科手术，同时为来自超过 100 个不同国家/地区的患者提供服务，这使其成为全球最大的儿童医疗中心之一。 波士顿儿童医院（Boston Children's Hospital）是全球知名的儿童医院，多年蝉联全美儿童医院综合排名第一/第二，小儿癌症全美第一/第二。此外，在儿童心脏、儿童神经、儿童骨科、儿童内分泌、儿童泌尿、儿童肺科等领域也稳居全美前列。 排名第一--神经内科和神经外科 《美国新闻与世界报道》将波士顿儿童医院的神经病学/神经外科部门评为全美最佳部门之一。 作为全球规模最大、历史最悠久、经验最丰富的神经内科和神经外科部门，波士顿儿童医院治疗的神经系统疾病比世界上任何其他儿科项目都要多。可以为患有神经系统疾病的儿童提供专业的临床护理，并提供广泛而深入的专科护理，以提供最准确的诊断和最先进的治疗方法。 排名第一--胃肠道科和胃肠外科 胃肠病学、肝病学和营养学科排名第一。团队由儿科胃肠病学家、肝病学家、营养师、心理学家、临床医生和护士组成，致力于帮助患有常见或复杂的胃肠道、肝脏和营养问题的儿童，例如： 乳糜泻 慢性腹痛 便秘和腹泻 克罗恩病 胃食管反流 营养不良 运动障碍 短肠综合征 等等，无论孩子刚确诊，还是正在寻找针对新症状或持续症状的新方案，专家团队都可以为您提供帮助。 排名第二--肿瘤科 波士顿儿童医院丹娜法伯癌症研究院联合成立了丹娜法伯/波士顿儿童医院癌症中心，共同创建了综合性儿童癌症和血液病项目，提供世界一流癌症中心和国际知名儿童医院的综合专业护理。丹娜法伯癌症研究所和波士顿儿童医院两家医院通过人行天桥连接，丹娜法伯/波士顿儿童医院提供的专家、治疗方案和临床试验比世界上几乎任何其他治疗癌症和血液病的儿科中心都要多。为患有各种癌症和血液病的儿童提供无缝、综合的医疗团队，包括最罕见和最复杂的病例。 儿童肾病 波士顿儿童医院被公认为治疗婴儿、儿童和青少年肾功能障碍的顶级中心之一，并被《美国新闻与世界报道》评为最好的儿童医院之一，在护理患有慢性肾病的幼儿方面享有国际声誉。对于患有复杂泌尿道异常、侵袭性肾病综合征或慢性肾炎、复杂肾结石以及导致电解质失衡的罕见遗传性肾病的儿童方面也拥有丰富的经验。此外，对于需要长期透析或肾移植的终末期肾病 (ESRD) 儿童方面也处于领先地位。 项目和服务 终末期肾病项目 普通肾脏病治疗计划 高血压计划 肾脏遗传学诊所 肾结石计划 肾脏移植计划 中主动脉综合征和肾血管性高血压中心 研究与创新 波士顿儿童肾脏病科拥有规模最大、历史最悠久的以儿科肾脏病为中心的研究项目之一。最新的研究包括： 肾脏移植研究项目，开展实验室研究，旨在开发更安全、更有效的免疫抑制方案 一项强有力的研究计划，旨在调查肾病综合征、终末期肾病和肾结石的遗传原因，目的是为每位患者制定个性化的治疗方案 一项多学科项目，引领开发针对中主动脉综合征和其他复杂高血压病因的新疗法 再生生物学团队致力于了解导致大多数慢性肾病的发育异常 药物基因组学小组，开发针对儿童基因差异的药物 波士顿儿童医院享誉全球,它的出色表现不仅在于其手术创新以及新的治疗方法的发现，还在于为儿童提供高质量的医疗并给予儿童高度的关注和关爱。 一、被全球公认为儿童肿瘤最好的医院 二、拥有两个世界上最大的儿童癌症和血液病研究项目 三、拥有众多世界知名的医师和科学家 四、基于研究的基础之上的治疗，为患者带来最新的治疗技术 五、为患有疑难和病情反复的患者提供众多临床试验、特殊治疗及高科技资源，以提高其治疗效果 六、多学科治疗，包括儿童血液学家、肿瘤学家、外科手术专家、影像学专家、病理学家、干细胞移植医生和心理学家共同会诊 七、制定儿童综合性的医疗护理计划，照顾到患有癌症和血液疾病的儿童各个方面的健康，给予儿童高程度的关注和关爱。 2024~2025世界最佳儿童医院排名（按字母顺序） 无论孩子患了什么病，这些医院都是全球治疗中名列前茅的！--美国USnews 为了能够给孩子们提供优质医疗照护，帮助迷茫的父母们寻求更为合适的医疗机构，美国三大新闻杂志之一的《美国新闻与世界报道》（U.S News & World Report）每年都会发布年度美国最佳儿童医院排行榜，至今已连续18年。全球肿瘤医生网医学部大家整理编译，希望给迷茫中的父母照亮希望之光。 2024-25年的排名是通过向近88家医院发送的临床调查收集的数据创建的，并向超过数千名儿科专家的医生进行了声誉调查。 波士顿儿童医院 在新生儿科、神经内科和神经外科以及泌尿科领域名列前茅。 辛辛那提儿童医院 在癌症、胃肠病学和胃肠外科以及肺病学和肺外科方面名列前茅。 奥罗拉科罗拉多儿童医院 在糖尿病和内分泌科领域排名第一。 洛杉矶儿童医院 在骨科和泌尿科领域名列前茅。 费城儿童医院 在糖尿病与内分泌学以及骨科领域名列前茅。 华盛顿特区儿童国家医院 在新生儿科领域排名第一。 俄亥俄州哥伦布市全国儿童医院 在胃肠病学和胃肠外科以及肾脏病学方面名列前茅。 圣地亚哥拉迪儿童医院 在心脏病学和心脏外科领域排名第一。 华盛顿州西雅图儿童医院 在肾脏病学领域排名第一。 休斯顿德克萨斯儿童医院 在心脏病学、心脏外科和肾脏病学方面名列前茅。 2024~2025世界最佳儿童癌症医院排名TOP10 排名 医院名称 1 辛辛那提儿童医院医疗中心 2 波士顿儿童医院 3 费城儿童医院 4 德克萨斯儿童医院 5 儿童国家医院 6 洛杉矶儿童医院 7 圣犹达儿童研究医院 8 科罗拉多儿童医院 9 全国儿童医院 10 西雅图儿童医院 2024~2025十大儿童专科世界排名TOP1 专科 医院名称 癌症 辛辛那提儿童医院 心脏病学和心脏外科 德克萨斯儿童医院 糖尿病和内分泌 费城儿童医院 胃肠病学和胃肠外科 辛辛那提儿童医院 新生儿科 波士顿儿童医院 肾脏科 德克萨斯儿童医院 神经科和神经外科 波士顿儿童医院 骨科 费城儿童医院 肺外科 辛辛那提儿童医院 泌尿科 波士顿儿童医院 参考资料：https://health.usnews.com/health-news/best-childrens-hospitals/articles/best-childrens-hospitals-honor-roll-and-overview