An open-label study to collect safety and effectiveness information on long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy who have completed prior studies with vamorolone (The GUARDIAN Study) - SNT-IV-VAM-011

开始日期2024-12-11

申办/合作机构

Santhera Pharmaceuticals Holding AG

NCT06713135

/ Recruiting临床4期

An Open-label Study to Collect Safety and Effectiveness Information on Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy Who Have Completed Prior Studies With Vamorolone

This study aims to assess safety and effectivness of long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy (DMD) who have completed prior studies with vamorolone.

开始日期2024-11-10

申办/合作机构-

NCT06689527

/ Completed临床1期

An Open-label, Single-arm Study to Evaluate the CYP3A4 Induction Potential of Vamorolone on the Pharmacokinetics of Midazolam (a Sensitive CYP3A4 Probe) in Healthy Subjects.

An open study to learn whether the study medication (vamorolone) is able to affect certain processes in the liver participating in the breakdown of drugs. This will be done by measuring the blood levels of midazolam alone and in combination with vamorolone in healthy participants.

开始日期2024-08-13

申办/合作机构-

100 项与伐莫洛龙相关的临床结果

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100 项与伐莫洛龙相关的转化医学

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项与伐莫洛龙相关的文献（医药）

2025-11-01·STEROIDS

Mineralocorticoid receptor antagonism of vamorolone: Evidence from LIONHEART and VISION-DMD clinical trials

Article

作者: Damsker, Jesse ; Hagerty, Laura ; Hoffman, Eric P ; Linden, Aki ; Gresko, Ekaterina ; Guglieri, Michela ; de Vera, Ana ; Berglund, Anders ; Dang, Utkarsh J ; Hasham, Shabir ; Clemens, Paula R ; Dutreix, Catherine ; Tobin, Rebecca ; Wahbi, Karim ; Hathout, Yetrib

The effect of vamorolone, the first dissociative corticosteroid for Duchenne muscular dystrophy (DMD), at the mineralocorticoid receptor (MR) was investigated in the Phase 1 mechanistic LIONHEART study and using serum samples from boys with DMD in the pivotal VISION-DMD trial. In LIONHEART, 30 healthy adult males were randomized 1:1:1 to vamorolone 20 mg/kg, eplerenone 200 mg, or no treatment arms. A fludrocortisone challenge was administered between -9 h and 24 h after treatment. The LIONHEART primary outcome was urinary Na⁺/K⁺ ratio; additional outcomes were pharmacokinetics, urine Na⁺ and K⁺ concentrations, and safety. In VISION-DMD, boys with DMD aged 4-7 years were treated with vamorolone 2 or 6 mg/kg/d for 48 weeks or with prednisone 0.75 mg/kg/d or placebo for 24 weeks followed by vamorolone 2 or 6 mg/kg/d for 20 weeks following a 4-week washout. Serum sample analysis from VISION-DMD used the SomaScan® 7 K assay. In LIONHEART, vamorolone reversed the decrease in urinary Na⁺/K⁺ ratio induced by fludrocortisone, confirming vamorolone MR antagonism. The maximum MRA effect of vamorolone was observed at 4-6 h post dose and was detectable until approximately 10 h post dose. Vamorolone reversed fludrocortisone induced Na⁺ retention with no evidence of decreased potassium excretion. Vamorolone 20 mg/kg was well tolerated, and results were consistent with known PK parameters. The VISION-DMD results showed vamorolone-specific increases in renin serum levels, as well as klotho, and calcium carrier proteins fetuin A and B, consistent with an MR antagonist effect. The available data confirm the MR antagonistic effect of vamorolone in humans. LIONHEART: NCT06649409; VISION-DMD: NCT03439670.

2025-10-01·BRAIN & DEVELOPMENT

Duchenne muscular dystrophy: Evolving therapeutic strategies and multidimensional evaluation approaches

Review

作者: Komaki, Hirofumi

Duchenne muscular dystrophy (DMD) is a severe X-linked muscular disorder caused by the absence of dystrophin. Standard therapies prolong survival; however, there is an increasing need for disease-modifying approaches and sensitive evaluation tools. This review presents a summary of the recent advances in DMD therapeutic strategies and multidimensional evaluation approaches, emphasizing their clinical applicability and future perspectives. In this study, a comprehensive review of the current literature was conducted focusing on mutation-specific therapies (exon skipping and gene therapy), non-mutation-specific pharmacological interventions (steroids, vamorolone, and histone deacetylase inhibitors), and emerging modalities such as cell-based therapy. The clinical outcome measures used across the disease stages were also examined, ranging from functional motor tests and imaging biomarkers to digital endpoints and quality of life (QOL) indices. Exon skipping and microdystrophin gene therapies have shown promising results in restoring partial dystrophin expression. Vamorolones and givinostat are alternative drugs with improved safety profiles. Time-based motor tests (such as time to sand, 10 m run) and digital biomarkers (Stride Velocity 95th Centile) enable the earlier detection of disease progression. Natural history data, including large registry-based datasets, enhance the interpretation of clinical trials. Patient-reported outcomes and disease-specific QOL measures, such as the DMD-QoL, are increasingly being incorporated to assess meaningful treatment benefits. The therapeutic landscape of DMD is evolving toward combination and personalized approaches. Multidimensional stage-specific evaluations are essential to capture functional changes and their therapeutic effects. Ongoing research on gene-, pharmacological-, and cell-based therapies, coupled with robust outcome assessments, may further improve patient care and QOL.

2025-10-01·JOURNAL OF PHARMACY TECHNOLOGY

Vamorolone Versus Traditional Glucocorticoids in Duchenne Muscular Dystrophy: A Review and Meta-Analysis of Efficacy and Safety

Review

作者: Jahanzeb, Ahmed ; Javed, Dawood ; Jajja, Faran Ahmed ; Javed, Abdullah

Objective: This meta-analysis evaluates the efficacy and safety of vamorolone, a dissociative glucocorticoid, compared with traditional glucocorticoids in treating Duchenne muscular dystrophy (DMD), aiming to assess its potential as a safer alternative with comparable therapeutic benefits. Data Sources: A systematic search was conducted in PubMed (MEDLINE), Embase, and Web of Science from inception till June 2024. Search terms included (Vamorolone) AND (Corticosteroids OR Glucocorticoids OR Prednisone) AND (Duchenne Muscular Dystrophy OR related terms). Only randomized controlled trials (RCTs) and observational studies were included, with no language restrictions. Study Selection and Data Extraction: From 276 identified reports, 135 were screened after duplicate removal, and 30 underwent full-text review. Five studies (2 RCTs, 2 observational, 1 nonrandomized trial) involving 370 patients were included. Two reviewers independently extracted data on efficacy (TTSTAND, TTRW, TTCLIMB velocities) and safety (height percentile, body mass index-z score, osteocalcin, P1NP) using Microsoft Excel, resolving discrepancies through consensus. Data Synthesis: Vamorolone significantly improved TTSTAND (MD = −0.03, 95% confidence interval [CI] = −0.06 to −0.004, P = .02), TTRW (MD = −0.11, 95% CI = −0.22 to −0.01, P = .04), and TTCLIMB velocities (MD = −0.04, 95% CI = −0.08 to −0.003, P = .03). Safety outcomes showed enhanced height percentile (MD = 16.28, 95% CI = 6.31–26.25, P = .001) and bone biomarkers (osteocalcin: MD = 15.68, P < .00001; P1NP: MD = 158.34, P < .00001), with no significant body mass index-z score difference. Conclusions: Vamorolone offers comparable efficacy with traditional glucocorticoids in DMD, with improved motor function and safer profiles in growth and bone health, suggesting its potential to transform DMD management.

247

项与伐莫洛龙相关的新闻（医药）

2025-10-09

·GlobeNewswire-Health Care

Santhera Secures Agreement with Biomedica for the Distribution of AGAMREE® (Vamorolone) in Russia

Pratteln, Switzerland, October 9, 2025 – Santhera Pharmaceuticals (SIX: SANN) announces the signing of an exclusive agreement with Biomedica for the distribution of AGAMREE® (vamorolone) in Russia, for the treatment of Duchenne muscular dystrophy (DMD) in patients four years of age and older. Under the terms of the agreement, Santhera will receive a percentage of net sales, in line with previous distribution agreements, with sales expected to begin in Q1 2026. This collaboration will enable access to AGAMREE for DMD patients across the country, facilitated through regional and national managed access programs. There are approximately 1,400 patients living with DMD in Russia [1]. Dario Eklund, Chief Executive Officer of Santhera, said: “This agreement will enable more children to access this important DMD treatment, and is the latest in a series of global agreements secured in 2025. We look forward to working closely with Biomedica in the months ahead.” Oleg Parosin, Founder and Chief Executive Officer of Biomedica added: “Our deep expertise in neurology and DMD, particularly with therapies that complement AGAMREE, combined with our track record of successful partnerships with leading EU and US companies, positions us as a strong and trusted partner for Santhera in Russia. We look forward to working together to accelerate access and ensure the availability of AGAMREE from early next year, enabling even more patients to benefit from this essential therapy.” About AGAMREE® (vamorolone) AGAMREE is a novel drug with a mode of action based on binding to the same receptor as glucocorticoids but modifying its downstream activity. Moreover, it is not a substrate for the 11-β-hydroxysteroid dehydrogenase (11β-HSD) enzymes that may be responsible for local drug amplification and corticosteroid-associated toxicity in local tissues [2-5]. This mechanism has shown the potential to ‘dissociate’ efficacy from steroid safety concerns and therefore AGAMREE is positioned as a dissociative anti-inflammatory drug and an alternative to existing corticosteroids, the current standard of care in children and adolescent patients with DMD [2-5]. In the pivotal VISION-DMD study, AGAMREE met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment and showed a good safety and tolerability profile [2, 5]. The most commonly reported side effects were cushingoid features, vomiting, weight increase and irritability. Side effects were generally of mild to moderate severity. Currently available data show that AGAMREE, unlike corticosteroids, has no restriction of growth [6] and no negative effects on bone metabolism as demonstrated by normal bone formation and bone resorption serum markers [7]. ▼ This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions. References:[1] Gordey Foundation [2] Dang UJ et al. (2024) Neurology 2024;102:e208112. doi.org/10.1212/WNL.0000000000208112. Link.[3] Guglieri M et al (2022). JAMA Neurol. 2022;79(10):1005-1014. doi:10.1001/jamaneurol.2022.2480 Link. [4] Liu X et al (2020). Proc Natl Acad Sci USA 117:24285-24293[5] Heier CR et al (2019). Life Science Alliance DOI: 10.26508[6] Ward et al., WMS 2022, FP.27 - Poster 71. Link.[7] Hasham et al., MDA 2022 Poster presentation. Link. About SantheraSanthera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular diseases with high unmet medical need. The Company has an exclusive license from ReveraGen for all indications worldwide to AGAMREE® (vamorolone), a dissociative steroid with novel mode of action, which was investigated in a pivotal study in patients with Duchenne muscular dystrophy (DMD) as an alternative to standard corticosteroids. AGAMREE for the treatment of DMD is approved in the U.S. by the Food and Drug Administration (FDA), in the EU by the European Commission (EC), in the UK by the Medicines and Healthcare products Regulatory Agency (MHRA), in China by the National Medical Products Administration (NMPA), in Hong Kong by the Department of Health (DoH) and Canada by Health Canada. Santhera has out-licensed rights to AGAMREE for North America to Catalyst Pharmaceuticals and for China and certain countries in Southeast Asia to Sperogenix Therapeutics. For further information, please visit www.santhera.com. AGAMREE® is a trademark of Santhera Pharmaceuticals. About BiomedicaBiomedica is a pharmaceutical company specializing in the commercialization of rare disease, innovative, and breakthrough therapies in underserved and emerging markets. With a strong presence across Central and Eastern Europe (CEE), the Commonwealth of Independent States (CIS), Turkey, and Israel, Biomedica partners with leading biotechnology and pharmaceutical innovators to bring novel treatments to patients. Its key therapeutic areas include rare diseases, neurology, haematology, oncology, hepatology and ophthalmology. Leveraging its regional expertise and established market access capabilities, Biomedica is committed to ensuring the timely access and availability of innovative therapies for patients with high unmet medical needs. For further information please contact: SantheraCatherine Isted, Chief Financial Officer: IR@santhera.com ICR Healthcare: Santhera@icrhealthcare.com BiomedicaStan Filippin, Head of Business Development: bd@biomedica.bio Disclaimer / Forward-looking statements This communication does not constitute an offer or invitation to subscribe for or purchase any securities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking statements concerning the Company and its business. Such statements involve certain risks, uncertainties and other factors which could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements. # # # Attachment 251009 Biomedica distirbution agreement_Final

引进/卖出上市批准

2025-10-06

·GlobeNewswire-Health Care

Santhera Announces Approval in Canada for AGAMREE® (Vamorolone) as a Treatment for Duchenne Muscular Dystrophy

Health Canada approved AGAMREE for the treatment of Duchenne muscular dystrophy in patients aged 4 years and older This approval makes AGAMREE the first and only approved therapy for DMD in Canada October 3, 2025 – Santhera Pharmaceuticals (SIX: SANN) today notes that Health Canada has approved AGAMREE® (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older following Priority Review, marking the first approved treatment for the disease in Canada. The Canadian Neuromuscular Disease registry estimates that there are more than 800 boys and young men living with DMD in Canada and coping with its debilitating symptoms. Kye Pharmaceuticals holds exclusive Canadian commercial rights to AGAMREE for the treatment of DMD and potentially other indications, following the agreement of a sub-license with Santhera’s commercialization partner Catalyst Pharmaceuticals, Inc. (“Catalyst”) in July 2024. Santhera will receive royalties on Canadian sales and these sales will also contribute towards North American sales milestones payable to Santhera from its partner Catalyst. This is the sixth independent approval a by local health authority, following positive regulatory decisions in the U.S., Europe, the UK, China and Hong Kong. Duchenne muscular dystrophy (DMD) is a rare inherited X-chromosome-linked disease, which almost exclusively affects males. DMD is characterized by inflammation which is present at birth or shortly thereafter. Inflammation leads to fibrosis of muscle and is clinically manifested by progressive muscle degeneration and weakness. Major milestones in the disease are the loss of ambulation, the loss of selffeeding, the start of assisted ventilation, and the development of cardiomyopathy. DMD reduces life expectancy to before the fourth decade due to respiratory and/or cardiac failure. Corticosteroids are the current standard of care for the treatment of DMD. AGAMREE is a novel drug with a mode of action based on binding to the same receptor as glucocorticoids but modifying its downstream activity. Moreover, it is not a substrate for the 11-β-hydroxysteroid dehydrogenase (11β-HSD) enzymes that may be responsible for local drug amplification and corticosteroid-associated toxicity in local tissues [1-4]. This mechanism has shown the potential to ‘dissociate’ efficacy from steroid safety concerns and therefore AGAMREE is positioned as a dissociative anti-inflammatory drug and an alternative to existing corticosteroids, the current standard of care in children and adolescent patients with DMD [1-4]. In the pivotal VISION-DMD study, AGAMREE met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment and showed a good safety and tolerability profile [1, 4]. The most commonly reported side effects were cushingoid features, vomiting, weight increase and irritability. Side effects were generally of mild to moderate severity. Currently available data show that AGAMREE, unlike corticosteroids, has no restriction of growth [5] and no negative effects on bone metabolism as demonstrated by normal bone formation and bone resorption serum markers [6]. References: [1] Dang UJ et al. (2024) Neurology 2024;102:e208112. doi.org/10.1212/WNL.0000000000208112. Link. [2] Guglieri M et al (2022). JAMA Neurol. 2022;79(10):1005-1014. doi:10.1001/jamaneurol.2022.2480. Link. [3] Liu X et al (2020). Proc Natl Acad Sci USA 117:24285-24293 [4] Heier CR et al (2019). Life Science Alliance DOI: 10.26508 [5] Ward et al., WMS 2022, FP.27 - Poster 71. Link. [6] Hasham et al., MDA 2022 Poster presentation. Link. Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular diseases with high unmet medical need. The Company has an exclusive license from ReveraGen for all indications worldwide to AGAMREE® (vamorolone), a dissociative steroid with novel mode of action, which was investigated in a pivotal study in patients with Duchenne muscular dystrophy (DMD) as an alternative to standard corticosteroids. AGAMREE for the treatment of DMD is approved in the U.S. by the Food and Drug Administration (FDA), in the EU by the European Commission (EC), in the UK by the Medicines and Healthcare products Regulatory Agency (MHRA), in China by the National Medical Products Administration (NMPA) and Hong Kong by the Department of Health (DoH). Santhera has out-licensed rights to AGAMREE for North America to Catalyst Pharmaceuticals and for China and certain countries in Southeast Asia to Sperogenix Therapeutics. Kye is a leading growth-stage Canadian specialty pharmaceutical company committed to bringing value to Canadians by identifying, licensing, and commercializing novel prescription medicines that may not otherwise be available to patients across Canada. With a growing pipeline of innovative medicines, Kye’s portfolio spans a range of therapeutic areas including cardiology, psychiatry, pediatrics, rare diseases, neuromuscular, hematology, and neurology. Kye Pharmaceuticals is a private company, founded in Canada and focused exclusively on the healthcare needs of patients in Canada. Kye aims to provide medicines that deliver better outcomes to our partners, Canadian healthcare professionals, and most importantly, patients across Canada. Kye, headquartered in Mississauga, Ontario was recognized on the Globe & Mail 2025 Canada’s Top Growing Companies list. The content above comes from the network. if any infringement, please contact us to modify.

上市批准引进/卖出优先审批临床结果

2025-10-06

·Pharmaceutical Technology

Health Canada approves Santhera’s Agamree to treat DMD

Agamree has secured approvals from several countries for Duchenne muscular dystrophy treatment. Credit: MY STOCKERS/Shutterstock.com. Health Canada has approved Santhera Pharmaceuticals’ Agamree (vamorolone) to treat Duchenne muscular dystrophy (DMD) in individuals aged four years and above. Agamree is the first approved therapy for DMD in the country. The decision follows a priority review and is supported by the Phase IIb VISION-DMD trial data, along with safety data from four open-label trials, including extensions. The studies evaluated the drug at doses between 2 to 6 mg/kg/day over periods up to 48 months. Agamree’s efficacy was on par with traditional corticosteroids, but with fewer adverse effects, particularly regarding bone health, growth and behaviour. In July 2024, Kye Pharmaceuticals secured exclusive commercial rights for Agamree in Canada, following a sub-licence agreement with Santhera’s commercialisation partner, Catalyst Pharmaceuticals. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence Kye Pharmaceuticals president and CEO John McKendry stated: “This Health Canada approval reflects Kye Pharmaceuticals’ commitment to advancing treatments for rare diseases and serving the unmet needs of Canadian patients. “We remain committed to working alongside the DMD community and with provincial drug plans and private insurers across Canada to ensure Agamree is accessible to individuals living with DMD.” Santhera will obtain royalties from Canadian sales, which will also contribute to the sales milestones from Catalyst for the North American market. The company has out-licensed the drug’s rights to Sperogenix Therapeutics for the Chinese market and certain Southeast Asian countries. During VISION-DMD, the drug met the primary goal by demonstrating an improvement in the time to stand velocity after 24 weeks of treatment when compared to a placebo, along with a favourable tolerability and safety profile. Santhera’s focus is on developing and commercialising treatments for rare neuromuscular conditions that lack adequate medical solutions. It holds an exclusive global licence from ReveraGen for all indications related to Agamree. The US Food and Drug Administration, China’s National Medical Products Administration, Hong Kong’s Department of Health, the European Commission and the UK Medicines and Healthcare products Regulatory Agency also approved Agamree for DMD treatment. Santhera previously signed an exclusive agreement with Ikris Pharma Network to distribute Agamree in India. Pharmaceutical Technology Excellence Awards - The Benefits of Entering Gain the recognition you deserve! The Pharmaceutical Technology Excellence Awards celebrate innovation, leadership, and impact. By entering, you showcase your achievements, elevate your industry profile, and position yourself among top leaders driving pharmaceutical advancements. Don’t miss your chance to stand out—submit your entry today! Nominate Now

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KEGG	Wiki	ATC	Drug Bank
D11000	伐莫洛龙	-	DB15114

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适应症	国家/地区	公司	日期
杜氏肌营养不良症	美国	Catalyst Pharmaceuticals, Inc.	2023-10-26

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适应症	最高研发状态	国家/地区	公司	日期
贝克型肌营养不良	临床2期	美国	ReveraGen BioPharma, Inc.	2022-07-07
贝克型肌营养不良	临床2期	美国	Santhera Pharmaceuticals Holding AG	2022-07-07
贝克型肌营养不良	临床2期	意大利	Santhera Pharmaceuticals Holding AG	2022-07-07
贝克型肌营养不良	临床2期	意大利	ReveraGen BioPharma, Inc.	2022-07-07
小儿溃疡性结肠炎	临床2期	-	ReveraGen BioPharma, Inc.	2020-09-01
囊性纤维化	临床前	美国	ReveraGen BioPharma, Inc.	2016-05-25
多发性硬化症	临床前	美国	ReveraGen BioPharma, Inc.	2016-05-25
炎症性肠病	临床前	美国	ReveraGen BioPharma, Inc.	2016-05-04
类风湿关节炎	临床前	美国	ReveraGen BioPharma, Inc.	2016-04-22
哮喘	临床前	美国	ReveraGen BioPharma, Inc.	2016-04-10

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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


NCT06649409 (LIONHEART, CTgov)	临床1期	-	30	Fludrocortisone+Eplerenone	齋簾夢餘選觸窪築餘觸(衊餘餘製鏇遞齋鹹顧衊) = 艱顧襯願網壓憲築遞簾壓衊製觸獵範觸鹽艱襯 (鏇窪簾糧膚鏇構構夢獵, 1.290309) 更多	-	2025-09-11
MDA2025	N/A	杜氏肌营养不良症	100	Vamorolone	製夢繭糧範積遞繭觸鑰(範鹹夢鹽糧鏇顧簾選獵) = 壓壓夢夢糧蓋醖鹽膚糧醖憲鏇願鏇夢壓艱衊醖 (遞簾襯繭願願鏇積襯鬱 ) 更多	积极	2025-03-16
NCT03439670 (VISION-DMD, MDA2025)	临床2期	杜氏肌营养不良症	118	Vamorolone 2 mg/kg/day	鹹蓋網鹽蓋願膚繭膚蓋(鹽糧鬱艱齋憲鹹製蓋積) = 鑰蓋鹽淵廠糧淵醖鹹願憲夢齋鏇願觸膚廠衊遞 (獵鹽積構鬱鹹艱製衊積 ) 更多	积极	2025-03-16
				Vamorolone 6 mg/kg/day	鹹蓋網鹽蓋願膚繭膚蓋(鹽糧鬱艱齋憲鹹製蓋積) = 鑰構觸鹽壓膚膚醖簾遞憲夢齋鏇願觸膚廠衊遞 (獵鹽積構鬱鹹艱製衊積 ) 更多
NCT05185622 (VBP15-006, MDA2025)	临床2期	杜氏肌营养不良症	54	Vamorolone 6 mg/kg/day (7-<18 years CS-treated)	繭蓋範鹽壓築簾構膚衊(餘獵觸艱壓壓淵製膚範) = Dose-dependent increases in adverse events were seen across all age groups; none led to study discontinuation or death 壓鑰鑰鏇鑰衊膚遞鏇窪 (齋遞構構網鏇觸襯繭鹹 ) 更多	积极	2025-03-16
LIONHEART (GlobeNewswire) 人工标引	N/A	-	30	AGAMREE	鹹蓋願簾壓選襯繭繭夢(繭糧鹽鏇簾壓醖淵衊製) = It demonstrated a statistically significant increase in the urinary sodium/potassium ratio in the vamorolone arm compared to placebo (p<0.0001) following a fludrocortisone challenge. 壓築觸鹹餘鑰窪構簾鑰 (顧網鏇簾繭壓鹽築築憲 )	积极	2024-10-01
				placebo
NCT03439670 (VISION-DMD \| VBP15-004, Literature) 人工标引	临床2期	杜氏肌营养不良症	121	vamorolone 2 mg/kg/d	鬱觸窪顧鏇膚遞鹽築鏇(鏇製糧網願淵醖鏇糧廠) = 齋淵憲壓築淵願膚積鹽簾遞衊網窪繭製製選範 (膚壓艱積範鑰願製糧淵, 0.0135) 更多	积极	2024-03-12
				vamorolone 6 mg/kg/d	鬱觸窪顧鏇膚遞鹽築鏇(鏇製糧網願淵醖鏇糧廠) = 醖壓夢膚蓋製顧鑰蓋窪簾遞衊網窪繭製製選範 (膚壓艱積範鑰願製糧淵, 0.0138) 更多
NCT03439670 (VISION-DMD, MDA2024) 人工标引	临床2期	杜氏肌营养不良症	118	Vamorolone 2 mg/kg/day	範繭積鹽蓋餘襯簾獵獵(廠憲構窪餘鹹願餘觸繭) = 範觸艱簾襯遞膚鑰鹽繭鑰鏇醖醖網鹽糧範簾淵 (齋窪範構觸艱築遞鏇廠 ) 更多	不佳	2024-03-03
				Prednisone 0.75 mg/kg/day	範繭積鹽蓋餘襯簾獵獵(廠憲構窪餘鹹願餘觸繭) = 鏇餘製鹽艱襯艱壓鬱繭鑰鏇醖醖網鹽糧範簾淵 (齋窪範構觸艱築遞鏇廠 ) 更多
NCT03439670 (FDA) 人工标引	临床2期	杜氏肌营养不良症	90	Placebo	鏇構鬱觸衊願觸餘餘艱(構襯獵淵積鬱網範廠鹽) = 鬱齋鹹壓獵鹹蓋願範壓構願遞簾顧選廠遞鹹繭 (鏇選壓壓憲範鏇積積膚 ) 更多	积极	2023-10-26
				AGAMREE 2 mg/kg/d	鏇構鬱觸衊願觸餘餘艱(構襯獵淵積鬱網範廠鹽) = 獵鏇憲齋觸餘鹹遞膚鏇構願遞簾顧選廠遞鹹繭 (鏇選壓壓憲範鏇積積膚 ) 更多
NCT03439670 (Pubmed \| JAMA Neurol)	临床2期	杜氏肌营养不良症	133	Vamorolone 2 mg/kg per day	餘製齋齋積築網構獵網(範繭網艱積鑰簾淵醖選) = Bone turnover markers declined with prednisone but not with vamorolone 簾襯鏇糧艱範顧襯鹹鏇 (醖鬱餘醖憲壓夢遞醖簾 ) 更多	积极	2022-08-29
NCT03439670 (VISION-DMD \| VBP15-004 \| VBP15-004, CTgov)	临床2期	杜氏肌营养不良症	121	Vamorolone (Treatment Group 1)	鬱鏇窪鏇築簾鏇網襯艱(襯壓鹹構顧鑰襯積繭憲) = 夢簾鏇艱餘窪製鹽鏇網獵積襯築膚襯廠淵顧窪 (膚淵淵蓋範廠願襯鏇衊, 0.0628) 更多	-	2022-07-13
				Vamorolone (Treatment Group 2)	鬱鏇窪鏇築簾鏇網襯艱(襯壓鹹構顧鑰襯積繭憲) = 廠壓蓋選構壓繭壓淵窪獵積襯築膚襯廠淵顧窪 (膚淵淵蓋範廠願襯鏇衊, 0.0666) 更多