The Supplementation of Vitamin A for the Prevention of Asparaginase-Associated Pancreatitis in Children With Acute Lymphoblastic Leukemia: A Prospective Single-arm Clinical Trial

开始日期2024-11-01

申办/合作机构-

NCT06450925 / Not yet recruiting临床2期IIT

A Randomized Double Blinded Trial of Vitamin A Supplementation in Allogeneic Stem Cell Transplantation.

The investigators hypothesize that single oral high dose supplementation with vitamin A will reduce the incidence of moderate-severe chronic graft-versus-host disease (GVHD) compared with placebo.

开始日期2024-10-01

申办/合作机构

Cincinnati Children's Hospital Medical Center

NCT06508099 / Recruiting临床2期IIT

Randomized Study of Vitamin A and D Prophylaxis Before Allogeneic Related and Unrelated Hematopoietic Stem Cell Transplantation

The therapy under investigation is the addition of 300 000 IU of vitamin A and 100 000 IU of vitamin D before conditioning. The study will include patients with malignant diseases in hematologic response with indications for allogeneic transplantation with matched related or matched unrelated donor.

开始日期2024-05-15

申办/合作机构-

100 项与维生素A 相关的临床结果

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100 项与维生素A 相关的转化医学

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100 项与维生素A 相关的专利（医药）

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项与维生素A 相关的文献（医药）

2025-02-01·BIOMATERIALS

Local delivery of platelet-derived factors mitigates ischemia and preserves ovarian function through angiogenic modulation: A personalized regenerative strategy for fertility preservation

Article

作者: Yang, Heeseon ; Lee, Kangwon ; Song, Chae Young ; Shin, Jungwoo ; Lee, Jung Ryeol ; Chung, Nanum ; Kim, Ji Hyang ; Min, Hyewon ; Yang, Chungmo

As the most prominent and ideal modality in female fertility preservation, ovarian tissue cryopreservation, and transplantation often confront the challenge of ischemic damage and follicular loss from avascular transplantation. To surmount this impediment, we engineered a novel platelet-derived factors-encapsulated fibrin hydrogel (PFH), a paradigmatic biomaterial. PFH encapsulates autologous platelet-derived factors, utilizing the physiological blood coagulation cascade for precise local delivery of bioactive molecules. In our study, PFH markedly bolstered the success of avascular ovarian tissue transplantation. Notably, the quantity and quality of follicles were preserved with improved neovascularization, accompanied by decreased DNA damage, increased ovulation, and superior embryonic development rates under a Low-concentration Platelet-rich plasma-derived factors encapsulated fibrin hydrogel (L-PFH) regimen. At a stabilized point of tissue engraftment, gene expression analysis mirrored normal ovarian tissue profiles, underscoring the effectiveness of L-PFH in mitigating the initial ischemic insult. This autologous blood-derived biomaterial, inspired by nature, capitalizes on the blood coagulation cascade, and combines biodegradability, biocompatibility, safety, and cost-effectiveness. The adjustable properties of this biomaterial, even in injectable form, extend its potential applications into the broader realm of personalized regenerative medicine. PFH emerges as a promising strategy to counter ischemic damage in tissue transplantation, signifying a broader therapeutic prospect. (197 words).

2025-02-01·BIOMATERIALS

Tumor microenvironment ameliorative and adaptive nanoparticles with photothermal-to-photodynamic switch for cancer phototherapy

Article

作者: Tang, Ben Zhong ; Ding, Keke ; Zhang, Le ; Liang, Ping ; Feng, Guangxue ; Zhang, Di ; Ji, Chao ; Yu, Yuewen

The notorious tumor microenvironment (TME) usually becomes more deteriorative during phototherapeutic progress that hampers the antitumor efficacy. To overcome this issue, we herein report the ameliorative and adaptive nanoparticles (TPASIC-PFH@PLGA NPs) that simultaneously reverse hypoxia TME and switch photoactivities from photothermal-dominated state to photodynamic-dominated state to maximize phototherapeutic effect. TPASIC-PFH@PLGA NPs are designed by incorporating oxygen-rich liquid perfluorohexane (PFH) into the intraparticle microenvironment to regulate the intramolecular motions of AIE photosensitizer TPASIC. TPASIC exhibits a unique aggregation-enhanced reactive oxygen species (ROS) generation feature. PFH incorporation affords TPASIC the initially dispersed state, thus promoting active intramolecular motions and photothermal conversion efficiency. While PFH volatilization leads to nanoparticle collapse and the formation of tight TPASIC aggregates with largely enhanced ROS generation efficiency. As a consequence, PFH incorporation not only currently promotes both photothermal and photodynamic efficacies of TPASIC and increases the intratumoral oxygen level, but also enables the smart photothermal-to-photodynamic switch to maximize the phototherapeutic performance. The integration of PFH and AIE photosensitizer eventually delivers more excellent antitumor effect over conventional phototherapeutic agents with fixed photothermal and photodynamic efficacies. This study proposes a new nanoengineering strategy to ameliorate TME and adapt the treatment modality to fit the changed TME for advanced antitumor applications.

2025-01-01·Synthetic and Systems Biotechnology

Systematic metabolic engineering enables highly efficient production of vitamin A in Saccharomyces cerevisiae

Article

作者: Dong, Tianyu ; Wu, Nan ; Zhang, Chenglong ; Zhou, Xiao ; Lu, Shuhuan ; Xing, Shilong ; Xiao, Wenhai ; Wang, Ying ; Shi, Yi ; Yao, Mingdong ; Wang, Xinhui

Vitamin A is a micronutrient critical for versatile biological functions and has been widely used in the food, cosmetics, pharmaceutical, and nutraceutical industries. Synthetic biology and metabolic engineering enable microbes, especially the model organism Saccharomyces cerevisiae (generally recognised as safe) to possess great potential for the production of vitamin A. Herein, we first generated a vitamin A-producing strain by mining β-carotene 15,15'-mono(di)oxygenase from different sources and identified two isoenzymes Mbblh and Ssbco with comparable catalytic properties but different catalytic mechanisms. Combinational expression of isoenzymes increased the flux from β-carotene to vitamin A metabolism. To modulate the vitamin A components, retinol dehydrogenase 12 from Homo sapiens was introduced to achieve more than 90 % retinol purity using shake flask fermentation. Overexpressing POS5Δ17 enhanced the reduced nicotinamide adenine dinucleotide phosphate pool, and the titer of vitamin A was elevated by almost 46 %. Multi-copy integration of the key rate-limiting step gene Mbblh further improved the synthesis of vitamin A. Consequently, the titer of vitamin A in the strain harbouring the Ura3 marker was increased to 588 mg/L at the shake-flask level. Eventually, the highest reported titer of 5.21 g/L vitamin A in S. cerevisiae was achieved in a 1-L bioreactor. This study unlocked the potential of S. cerevisiae for synthesising vitamin A in a sustainable and economical way, laying the foundation for the commercial-scale production of bio-based vitamin A.

210

项与维生素A 相关的新闻（医药）

2024-10-23

·BioSpace

Topline Results from Alkeus Pharmaceuticals’ Study of Oral Gildeuretinol Demonstrate Significant Trend in Slowing GA Progression and Visual Function Improvement

Oral gildeuretinol demonstrated a statistically significant reduction in the geographic atrophy (GA) lesion growth rate of 15.3% from 6 to 24 months (p=0.047) in a pre-specified analysis. Gildeuretinol is the first investigational oral therapy to demonstrate a functional benefit in low luminance visual acuity (LLVA) over 24 months (p=0.031), a key secondary endpoint. Gildeuretinol showed a clinically meaningful reduction in GA lesion growth rate of 13.4% from baseline to 24 months (p=0.075), the study’s primary endpoint. Results indicate that slowing the formation of toxic vitamin A dimers could help address the high unmet need for oral therapies in multiple retinal diseases. Gildeuretinol demonstrated a favorable safety and tolerability profile. Among participants at risk of developing Choroidal Neovascularization (CNV) or wet AMD, only 11% in the gildeuretinol group experienced these adverse events compared to 44% in the placebo group, as reported by clinical investigators. CAMBRIDGE, Mass., Oct. 23, 2024 (GLOBE NEWSWIRE) -- Alkeus Pharmaceuticals, Inc., a biopharmaceutical company dedicated to preserving the sight of individuals impacted by retinal diseases, today announced that patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) treated with investigational oral therapy gildeuretinol (ALK-001) showed a statistically significant reduction in the GA lesion growth rate of 15.3% vs. placebo from 6 to 24 months (p=0.047). Gildeuretinol also demonstrated a statistically significant visual function improvement, showing 4.4 fewer letters lost (p=0.031) in low luminance visual acuity (LLVA) over 24 months. Gildeuretinol showed a clinically meaningful trend in slowing GA growth rate of 13.4% from baseline to 24 months (p=0.075), the study’s primary endpoint. A trend toward functional benefit in best corrected visual acuity (BCVA) was demonstrated with 3.3 fewer letters lost (p=0.099). Topline results of the SAGA study of gildeuretinol for the treatment of GA secondary to AMD were presented by Alexander Melamud, M.D., M.A., retina specialist and vitreoretinal surgeon with the Retina Group of Washington and a principal investigator in the SAGA study during the 128th Annual Meeting of the American Academy of Ophthalmology (AAO) held in Chicago October 18-21, 2024. “These data indicate a significant slowing of the growth rate of GA lesions, as well as reducing visual function decline,” said Seemi Khan, M.D., M.P.H., M.B.A., Chief Medical Officer of Alkeus Pharmaceuticals. “It takes a couple of months for gildeuretinol to replace vitamin A and achieve a therapeutic effect. Therefore, we are encouraged by the sensitivity analysis measuring the effect starting at six months of treatment. These data represent the first clinical demonstration that slowing vitamin A dimerization could be a target in the treatment of GA secondary to AMD. By slowing dimerization with gildeuretinol’s unique mechanism, we could reduce further damage to the retina at more advanced stages and possibly delay or prevent the onset of GA. We are excited by the potential of gildeuretinol as an oral therapy with functional benefits in GA, in addition to its potential to be the first therapy for Stargardt disease. We would like to thank the patients, investigators and trial sites for their participation in this study, as well as the National Institutes of Health National Eye Institute for providing funding for this study.” SAGA was a 24-month, double-masked, randomized, placebo-controlled trial to investigate the safety, pharmacokinetics, tolerability and efficacy in patients with GA secondary to AMD. The study enrolled 198 patients. The primary efficacy endpoint was the growth rate of GA lesions from baseline to 24 months as assessed by Fundus Autofluorescence (FAF). The first key secondary endpoint was the change in LLVA from baseline to 24 months. A favorable safety and tolerability pro demonstrated by gildeuretinol, consistent with prior clinical studies of gildeuretinol in Stargardt disease. Additionally, among participants at risk of developing Choroidal Neovascularization (CNV) or wet AMD, only 11% in the gildeuretinol group experienced these adverse events compared to 44% in the placebo group, as reported by the retina specialist investigators. “These data are meaningful and changing the conversation in the space about what might be possible with next generation treatments for patients with GA and earlier stages of AMD,” said Charles Wykoff, M.D., Ph.D., retina specialist and Director of Research at Retina Consultants of Texas. “While the primary endpoint was not met, the anatomic efficacy signal appears real, and most interestingly there appears to be a consistent signal of functional preservation with treatment. This patient population could benefit tremendously from a safe and effective systemic treatment option.” GA is a serious, progressive condition that causes irreversible loss of central vision. It is estimated that the median time of progression to severe visual impairment is slightly over six years. There is no oral therapy approved by the FDA to treat GA. The prevalence of GA in the United States is estimated to be over 1 million people, with 160,000 new cases occurring each year. “One of the most exciting aspects of these results is that gildeuretinol is the first oral medication to show a meaningful positive effect on low luminance visual acuity in macular degeneration,” Dr. Melamud said. “One of the first things patients lose in macular degeneration is low light vision, which can have a dramatic impact on daily living. The positive effect demonstrated on LLVA is significant. Based on these results, this therapy would definitely have a place in the overall strategy of treating AMD and slowing the decline observed in our patients. The favorable safety pro warrant treatment as early as possible.” About Gildeuretinol Acetate (ALK-001) Oral gildeuretinol acetate (ALK-001) is a new chemical entity designed to reduce the dimerization of vitamin A without modulating the visual cycle. In preclinical studies, gildeuretinol decreased vitamin A dimerization down to the normal rate and prevented retinal degeneration and loss of visual function in animals with Stargardt disease. A randomized, placebo-controlled, double-masked clinical trial of gildeuretinol in late-stage Stargardt patients (TEASE-1) showed clinically and statistically significant slowing of the growth of retinal lesions over two years of treatment. Additional clinical trials of gildeuretinol in Stargardt disease are ongoing. Gildeuretinol has received breakthrough therapy designation and orphan drug designation for Stargardt disease from the U.S. Food and Drug Administration. A study (SAGA) of gildeuretinol in 198 patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) demonstrated a meaningful trend in the reduction of lesion growth rate and demonstrated a functional benefit in low luminance visual acuity (LLVA). In studies, gildeuretinol demonstrated a favorable safety and tolerability profile. About Alkeus Pharmaceuticals Alkeus Pharmaceuticals, Inc. is a private biopharmaceutical company with headquarters in Cambridge, Mass., backed by institutional investors led by Bain Capital Life Sciences. Founded in 2010, Alkeus is developing therapies for serious diseases of the eye with high unmet need, with the purpose to protect the sight of individuals impacted by retinal diseases. Alkeus’ breakthrough-designated lead candidate, gildeuretinol acetate (ALK-001), is a new chemical entity currently being evaluated in clinical trials for the treatment of Stargardt disease and for geographic atrophy (GA) secondary to age-related macular degeneration (AMD). For further information, please contact: Media Relations media@alkeuspharma.com Website:

临床结果孤儿药突破性疗法

2024-10-21

·蒲公英Ouryao

药辅龙头公司突发安全事故，一员工不幸遇难

转自：企业公告整理：水晶 10月21日，安徽山河药用辅料股份有限公司公告称，10月19日14时许，公司发生一起安全事故，经初步调查，系交联羧甲纤维素钠车间员工清理酒精母液罐罐底浆料时发生意外事故。事故造成一名员工窒息，送医院抢救无效死亡。事故仍在调查中。 10月21日，安徽山河药用辅料股份有限公司（简称山河药辅）公告称，10月19日14时许，公司“新型药用辅料系列生产基地一期项目”（简称南区）发生一起一般安全事故，经初步调查，系交联羧甲纤维素钠车间员工清理酒精母液罐罐底浆料时发生意外事故。事故造成一名员工窒息，送医院抢救无效死亡。发生事故车间是位于南区的交联羧甲纤维素钠生产线。产量占公司总体业务比重较小，公司已经责令该车间自查整改，进行全面安全风险隐患排查。截止本公告披露日，事故仍在调查中。目前，公司整体系列生产基地和车间生产经营秩序正常。公开资料显示，山河药辅是专业从事新型药用辅料生产、研发和销售为一体的民营股份制企业，目前在国内药用辅料（固体制剂）行业处于龙头地位。其产品涵盖填充剂、黏合剂、崩解剂、润滑剂、包衣材料等常用口服固体制剂类药用辅料。 2024年药企发生安全事故的还有：河南沈丘县一药厂突发大火：8月19日晚河南沈丘县一药厂突发大火，火势凶猛，浓烟数公里外可见。附近居民称被巨响震醒。辽宁锦州九泰药业，2人死亡3人受伤：2024年5月12日0时25分，锦州鑫泰基精细化工有限公司（隶属于锦州九泰药业有限责任公司）A区车间发生安全事故起火。2时33分，现场明火被扑灭，两名被困人员经抢救无效死亡，另有三名伤者在医院接受救治，伤势轻微，无生命危险。金达威，3人死亡3人受伤：1月11日上午9时许，厦门金达威集团股份有限公司全资子公司厦门金达威维生素有限公司污水处理池空间发生一起闪爆事件。经初步调查，系金达威维生素在污水处理池上方施工安装遮阳棚过程中突发池内空间可燃气体闪爆，导致污水处理池及上方遮阳棚坍塌，事故造成现场施工人员3人死亡3人受伤送医救治。印度一家制药厂发生爆炸17人死亡：8月21日，印度安得拉邦一家制药厂当时约有190名工人正在上班，据报道该制药厂一个化学反应装置发生爆炸，附近村庄居民听到巨大爆炸声。事故发生后，数十辆消防车前往灭火，但现场烟雾弥漫，救援行动一度受阻。巴斯夫德国工厂发生爆炸：7月29日中午12点左右，巴斯夫路德维希港基地南部的一处工厂发生有机溶剂泄漏，随后发生火灾爆炸，爆炸发生后巴斯夫表示，正在通知客户，可以从公司库存中获得产品，产品数量将根据相关法律以公平合理的方式进行分配，截止8月16日，目前已知的受影响产品包括：维生素 A、维生素 E 和类胡萝卜素、DL-薄荷醇等产品。日本一药企发生火灾：1月9日，当天上午东京板桥区一家药品工厂发生溶剂泄漏并着火。目前已经确认有3人受伤，其中1人伤势严重。安全无小事，警钟长鸣！！！

2024-10-21

·PRNewswire

WAINZUA (eplontersen) recommended for approval in the EU by CHMP for the treatment of adults with polyneuropathy associated with hereditary transthyretin-mediated amyloidosis

- Recommendation based on NEURO-TTRansform Phase 3 results showing WAINZUA demonstrated consistent and sustained benefit improving neuropathy impairment and quality of life versus placebo CARLSBAD, Calif., Oct. 21, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that Ionis' and AstraZeneca's WAINZUA (eplontersen) has been recommended for approval by the Committee for Medicinal Products for Human Use (CHMP) in the European Union (EU) for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy, commonly referred to as hATTR-PN or ATTRv-PN. If approved by the European Commission, WAINZUA will be the only approved medicine in the EU for the treatment of ATTRv-PN that can be self-administered monthly via an auto-injector. The CHMP based its opinion on the positive NEURO-TTRansform Phase 3 trial which showed that through 66 weeks, patients treated with WAINZUA demonstrated consistent and sustained benefit on the co-primary endpoints of serum transthyretin (TTR) concentration and neuropathy impairment measured by modified Neuropathy Impairment Score +7 (mNIS+7), and key secondary endpoint of quality of life (QoL) on the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) versus external placebo. WAINZUA continued to demonstrate a favorable safety and tolerability profile throughout the NEURO-TTRansform trial. ATTRv-PN is a debilitating disease that leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade. WAINZUA is a once-monthly RNA-targeted medicine designed to reduce the production of TTR protein at its source. "Hereditary transthyretin-mediated amyloidosis with polyneuropathy remains a progressive and debilitating disease in Europe and other parts of the world, despite currently available medicines," said Brett P. Monia, Ph.D., chief executive officer, Ionis. "The CHMP recommendation is an important step toward making WAINZUA available in Europe, which, if approved, will be the only medicine in the EU for the treatment of transthyretin-mediated amyloidosis with polyneuropathy that can be self-administered monthly via an auto-injector. We are proud to partner with AstraZeneca whose global leadership and expertise positions our alliance to rapidly and effectively bring WAINZUA to many people living with hereditary transthyretin-mediated amyloidosis with polyneuropathy in Europe, pending the EMA's decision." In December 2023, eplontersen was approved in the U.S. for the treatment of ATTRv-PN, under the brand name WAINUA™ (eplontersen). WAINUA is now gaining approvals in additional countries worldwide, including Canada. As part of a global development and commercialization agreement, AstraZeneca and Ionis are commercializing WAINUA in the U.S. The companies are seeking regulatory approval in Europe and other parts of the world where AstraZeneca has exclusive rest of world commercialization and development rights. Eplontersen has also been granted Orphan Drug Designation in the U.S. and in the EU for the treatment of transthyretin-mediated amyloidosis (ATTR). Eplontersen is also currently being evaluated in the global CARDIO-TTRansform Phase 3 study for the treatment of adults with ATTR-CM. The trial is fully enrolled with more than 1,400 patients – making it the largest, most comprehensive study to date in this patient population. More information on the CARDIO-TTRansform study (NCT04136171) is available at . About TTR Amyloidosis ATTR is caused by the accumulation of liver-derived misfolded TTR protein in tissues, such as the heart and the peripheral nerves, causing organ damage and failure. ATTR then causes complications, leading to cardiovascular, neurological and renal diseases such as heart failure and chronic kidney disease. There are both hereditary (ATTRv) and non-hereditary (wild-type) forms of ATTR. ATTR is a rapidly progressive and fatal disease that requires timely recognition of symptoms. ATTR has several phenotypes including ATTR-cardiomyopathy (CM), which predominantly impacts the heart, potentially leading to heart failure, ATTR-polyneuropathy (PN), which predominantly affects the peripheral nervous system and mixed phenotype, where patients experience symptoms of both. Worldwide, there are an estimated 300,000 – 500,000 patients with ATTR-CM and about 10,000 – 40,000 patients with ATTRv-PN. About NEURO-TTRansform NEURO-TTRansform is a global, open-label, randomized trial evaluating the efficacy and safety of eplontersen in patients with ATTRv-PN at week 35, week 66 and week 85. The final analysis comparing eplontersen to an external placebo group was completed at week 66. All patients were then followed on treatment until week 85 and evaluated four weeks after the last dose in an end-of-trial assessment. Following treatment and the end-of-trial assessments, patients were eligible to enter an open-label extension study to continue receiving eplontersen once every four weeks or enter a 20-week post-treatment evaluation period, which is still ongoing. Full results from the NEURO-TTRansform trial were published in The Journal of the American Medical Association (JAMA) demonstrating the benefit of eplontersen across the spectrum of ATTRv-PN at 35, 66 and 85 weeks of treatment. About WAINZUA / WAINUA™ (eplontersen) Eplontersen is a once-monthly RNA-targeted medicine that provides upstream suppression of transthyretin (TTR) production and is designed to precisely target and reduce the production of TTR protein at its source in the liver. WAINZUA has been approved in the U.S. and Canada under the brand name WAINUA™ (eplontersen) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN. Please see link for full U.S. Prescribing Information. WAINZUA is not yet approved for any indication in Europe. U.S. INDICATION for WAINUA™ (eplontersen) WAINUA injection for subcutaneous use 45 mg is indicated for treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. IMPORTANT SAFETY INFORMATION for WAINUA™ (eplontersen) WARNINGS AND PRECAUTIONS Reduced Serum Vitamin A Levels and Recommended Supplementation WAINUA leads to a decrease in serum vitamin A levels. Supplement with recommended daily allowance of vitamin A. Refer patient to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur. ADVERSE REACTIONS Most common adverse reactions (≥9% in WAINUA-treated patients) were vitamin A decreased (15%) and vomiting (9%). Please see link to U.S. Full Prescribing Information for WAINUA. About Ionis Pharmaceuticals, Inc. For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has five marketed medicines and a leading pipeline in neurology, cardiology, and other areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit Ionis.com and follow us on X (Twitter) and LinkedIn. Ionis Forward-looking Statements This press release includes forward-looking statements regarding eplontersen, Ionis' business, and the therapeutic and commercial potential of Ionis' commercial medicines, additional medicines in development and technologies. Any statement describing Ionis' goals, expectations, financial or other projections, intentions, or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, including but not limited to those related to our commercial products and the medicines in our pipeline, and particularly those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended Dec. 31, 2023, and most recent Form 10-Q, which are on file with the SEC. Copies of these and other documents are available at . Ionis Pharmaceuticals® is a registered trademark of Ionis Pharmaceuticals, Inc. WAINUA™ is a trademark of AstraZeneca plc. Ionis Investor Contact: D. Wade Walke, Ph.DD. [email protected] 760-603-2331 Ionis Media Contact: Daria Binder [email protected] 760-603-4679 SOURCE Ionis Pharmaceuticals, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床3期临床结果孤儿药上市批准

100 项与维生素A 相关的药物交易

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KEGG	Wiki	ATC	Drug Bank
D06543	维生素A	A11CA01	DB00162

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10 条最早获批的记录,

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适应症	国家/地区	公司	日期
寻常鳞癣	日本	Sannova KK	1979-09-08
糠疹	日本	Sannova KK	1979-09-08
干眼症	-	-	-

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适应症	最高研发状态	国家/地区	公司	日期
维生素a缺乏症	临床申请批准	美国	-	2023-12-12

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


ESGO2024	N/A	葡萄胎 Î²-HCG	-	Vitamin A	遞淵構蓋膚醖蓋糧繭選(壓窪憲遞窪製醖壓選鬱) = 範觸醖觸製餘襯遞願觸網積淵鬱鹽淵構餘鬱鏇 (膚醖蓋構觸鹹襯餘築繭 )	积极	2024-03-10
Tandem Meetings ASTCT and CIBMTR2023	N/A	慢性移植物抗宿主病	-	High-dose oral vitamin A	願顧衊製願齋網糧壓築(窪蓋繭艱襯築衊構夢顧) = 襯範鹽鹽範鹽餘餘夢憲鬱餘艱艱鹽壓顧鬱鬱糧 (廠願選鬱繭憲鏇蓋範鑰 ) 更多	积极	2023-02-01
				Placebo	願顧衊製願齋網糧壓築(窪蓋繭艱襯築衊構夢顧) = 積選襯憲選鹽醖襯壓壓鬱餘艱艱鹽壓顧鬱鬱糧 (廠願選鬱繭憲鏇蓋範鑰 ) 更多
ASN2022	N/A	高钙血症	-	Vitamin A Toxicity	襯鹽觸窪顧遞鹹構齋衊(廠廠積衊窪齋廠鏇蓋獵) = 鹽壓餘選醖憲鏇鏇憲糧鏇窪願膚選獵觸鬱醖醖 (築觸衊遞鑰鹹範醖糧廠 )	-	2022-11-03
ARVO2022	N/A	年龄相关性黄斑变性	-	Low dose Vitamin A	觸鑰淵積積淵鬱積醖鑰(廠網齋襯夢網築鏇膚膚) = 窪顧觸膚夢遞夢齋獵鹹觸窪遞顧糧鏇選廠鏇觸 (壓積鏇製壓膚鏇繭遞糧, 6.5)	-	2022-05-01
				Low dose Vitamin A	觸鑰淵積積淵鬱積醖鑰(廠網齋襯夢網築鏇膚膚) = 鏇衊襯憲艱齋遞鏇觸襯觸窪遞顧糧鏇選廠鏇觸 (壓積鏇製壓膚鏇繭遞糧, 10.9)
NCT01203488 (Pubmed)	临床3期	支气管肺发育不良	807	Vitamin A therapy	壓齋醖壓築蓋築顧壓蓋(鹹鹽醖製蓋蓋顧壓鹽鏇): RR = 0.89 (95% CI, 0.8 ~ 0.99)	积极	2021-05-15
				Vitamin A (Control group)
Pubmed \| Pediatrics	N/A	支气管肺发育不良	188	Enteral water-soluble vitamin A	憲觸遞蓋醖觸製繭顧網(蓋鹹齋餘襯鹽淵選壓願) = 積鬱積繭憲鬱鏇獵鏇鑰觸淵齋鏇繭餘醖鏇範鏇 (糧鑰蓋壓鑰憲構簾鑰鑰 ) 更多	-	2021-01-01
				Placebo	憲觸遞蓋醖觸製繭顧網(蓋鹹齋餘襯鹽淵選壓願) = 醖鬱繭鑰糧艱鹹鑰積獵觸淵齋鏇繭餘醖鏇範鏇 (糧鑰蓋壓鑰憲構簾鑰鑰 ) 更多
NCT00514891 (Pubmed \| Pediatrics)	临床4期	-	7,587	Vitamin A supplementation	醖獵鬱憲糧簾網獵範蓋(齋艱顧鏇夢顧積蓋餘範): MRR = 0.91 (95% CI, 0.59 ~ 1.41)	-	2014-09-01
				vitamin A (Placebo)
NCT00168610 (Pubmed \| J Nutr)	临床4期	-	6,048	50,000 IU neonatal vitamin A	構遞觸網獵築願顧顧夢(鹹壓積醖願憲鑰窪壓遞) = 鑰廠繭選鑰鏇觸鹽觸膚鏇衊襯願獵壓積醖獵廠 (繭鑰鹹繭淵繭獵築衊選 )	-	2014-09-01
				25,000 IU neonatal vitamin A	構遞觸網獵築願顧顧夢(鹹壓積醖願憲鑰窪壓遞) = 蓋鏇壓糧淵範窪構選窪鏇衊襯願獵壓積醖獵廠 (繭鑰鹹繭淵繭獵築衊選 )
NCT00936091 (Pubmed \| Parasit Vectors)	N/A	-	250	Vitamin A supplementation	製衊觸願齋製鏇獵願獵(鏇鏇艱範廠繭選鹽願製) = 醖築選廠齋窪鑰繭鬱構糧廠鏇範窪憲夢醖遞觸 (積餘醖遞齋製選鬱窪壓 ) 更多	积极	2014-08-15
				vitamin A supplementation (Placebo)	製衊觸願齋製鏇獵願獵(鏇鏇艱範廠繭選鹽願製) = 壓選遞夢簾醖鹽積構鹽糧廠鏇範窪憲夢醖遞觸 (積餘醖遞齋製選鬱窪壓 ) 更多
Pubmed \| Nutrients	N/A	缺铁性贫血	250	Vitamin A supplementation	蓋鹽顧憲觸蓋壓觸衊鬱(構膚鹹襯築壓憲獵築簾) = 製壓鏇觸鑰願簾襯齋淵獵襯夢夢鏇齋簾構廠淵 (選鏇憲窪顧餘壓選鹹蓋, 28.3 ~ 40.2) 更多	积极	2013-12-31