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The alpha-1 antitrypsin deficiency market size shall grow during the forecast period (2023–2032) due to the launch of upcoming therapies and the increasing cases of the alpha-1 antitrypsin deficiency.
由于即将推出的疗法以及α-1抗胰蛋白酶缺乏症病例的增加,在预测期(2023-2032年)内,α-1抗胰蛋白酶缺乏症的市场规模将增长。
LAS VEGAS, Jan. 18, 2024 /PRNewswire/ -- DelveInsight's Alpha-1 Antitrypsin Deficiency Market Insights report includes a comprehensive understanding of current treatment practices, alpha-1 antitrypsin deficiency emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan]..
拉斯维加斯,2024年1月18日/PRNewswire/--DelveInsight的Alpha-1抗胰蛋白酶缺乏症市场见解报告包括对当前治疗实践、Alpha-1抗胰蛋白酶缺乏症新兴药物、个体疗法的市场份额以及2019年至2032年的当前和预测市场规模的全面了解,分为7MM[美国、EU4(德国、法国、意大利和西班牙)、英国和日本]。。
Key Takeaways from the Alpha-1 Antitrypsin Deficiency Market Report
Alpha-1抗胰蛋白酶缺乏症市场报告的关键要点
According to DelveInsight's analysis, the market size for AATD reached USD 1.1 billion in 2022 across the 7MM and is expected to grow with a significant CAGR by 2032.
根据DelveInsight的分析,AATD的市场规模在2022年达到11亿美元,达到700万美元,预计到2032年将以显著的复合年增长率增长。
DelveInsight's analysis reveals that the overall diagnosed prevalent population of AATD in the 7MM was reported as ~15 million in 2022.
DelveInsight的分析显示,据报道,2022年,700万AATD的总诊断流行人群约为1500万。
Leading alpha-1 antitrypsin deficiency companies such as Kamada Pharmaceuticals, Arrowhead Pharmaceuticals, Takeda, Mereo BioPharma, AstraZeneca, Vertex Pharmaceuticals, Inhibrx, Novo Nordisk, and others are developing novel AATD drugs that can be available in the AATD market in the coming years.
领先的α-1抗胰蛋白酶缺乏症公司,如Kamada Pharmaceuticals,Arrowhead Pharmaceuticals,Takeda,Mereo BioPharma,AstraZeneca,Vertex Pharmaceuticals,Inhibrx,Novo Nordisk等,正在开发新的AATD药物,这些药物可以在未来几年的AATD市场上买到。
The promising alpha-1 antitrypsin deficiency therapies in the pipeline include Inhaled Alpha 1-Antitrypsin (AAT), Fazirsiran (ARO-AAT/TAK-999), Alvelestat (MPH-966), VX-864, INBRX-101, Belcesiran (DCR-A1AT), and others.
正在开发的有前途的α-1抗胰蛋白酶缺乏症疗法包括吸入α-1-抗胰蛋白酶(AAT),法齐西兰(ARO-AAT/TAK-999),阿尔维列斯坦(MPH-966),VX-864,INBRX-101,Belcesiran(DCR-A1AT)等。
Understanding the underlying mechanisms of alpha-1 antitrypsin deficiency has progressed, paving the way for the development of therapeutic approaches targeting alternative pathways. Novel treatments such as Kamada Pharmaceutical's inhaled alpha 1-antitrypsin (AAT), Arrowhead Pharmaceuticals/Takeda's fazirsiran (ARO-AAT/TAK-999), Vertex Pharmaceutical's VX-864, and Mereo BioPharma/AstraZeneca's alvelestat (MPH-966) are emerging as potential long-term therapeutic agents.
了解α-1抗胰蛋白酶缺乏症的潜在机制已取得进展,为开发针对替代途径的治疗方法铺平了道路。Kamada Pharmaceutical的吸入性α1-抗胰蛋白酶(AAT),Arrowhead Pharmaceuticals/Takeda的fazirsiran(ARO-AAT/TAK-999),Vertex Pharmaceutical的VX-864和Mereo BioPharma/AstraZeneca的alvelestat(MPH-966)等新疗法正在成为潜在的长期治疗药物。
The approval of these innovative treatments is expected to reshape the alpha-1 antitrypsin deficiency market during the forecast period, enabling the entry of new players into the treatment landscape..
预计这些创新疗法的批准将在预测期内重塑α-1抗胰蛋白酶缺乏症市场,使新参与者能够进入治疗领域。。
Among the emerging therapies, Mereo BioPharma/AstraZeneca's alvelestat (MPH-966) is a neutrophil elastase enzyme inhibitor that helps protect people with alpha-1 antitrypsin deficiency by slowing progressive lung damage. It is expected to enter the US market by 2028 and is predicted to have a medium-fast uptake during the forecast period..
在新兴疗法中,Mereo BioPharma/AstraZeneca的alvelestat(MPH-966)是一种中性粒细胞弹性蛋白酶抑制剂,可通过减缓进行性肺损伤来帮助保护α-1抗胰蛋白酶缺乏症患者。预计它将于2028年进入美国市场,预计在预测期内将以中速增长。。
Discover which therapies are expected to grab the major AATD market share @ Alpha-1 Antitrypsin Deficiency Market Report
发现哪些疗法有望在α-1抗胰蛋白酶缺乏症市场报告中占据AATD的主要市场份额
Alpha-1 Antitrypsin Deficiency Overview
α-1抗胰蛋白酶缺乏症概述
Alpha-1 Antitrypsin Deficiency (AATD) is a hereditary autosomal codominant genetic disorder characterized by a shortage of the alpha-1 antitrypsin protein (AAT). This protein is crucial for safeguarding the lungs and liver from enzyme-induced damage, and its insufficiency can result in severe respiratory and liver-related complications.
α-1抗胰蛋白酶缺乏症(AATD)是一种遗传性常染色体显性遗传疾病,其特征是缺乏α-1抗胰蛋白酶蛋白(AAT)。这种蛋白质对于保护肺部和肝脏免受酶诱导的损伤至关重要,其不足可导致严重的呼吸和肝脏相关并发症。
The condition primarily arises from mutations in the SERPINA1 gene, responsible for producing the AAT protein, which shields the body from the potent enzyme neutrophil elastase..
这种情况主要是由SERPINA1基因突变引起的,该基因负责产生AAT蛋白,该蛋白可以保护身体免受中性粒细胞弹性蛋白酶的侵害。。
AATD encompasses symptoms such as breathlessness, persistent coughing, wheezing, and a diminished capacity for exercise. The emergence and intensity of these AATD symptoms can widely differ among individuals, even those sharing identical genetic mutations. While some individuals may exhibit no symptoms, others may face considerable health challenges.
AATD包括呼吸困难,持续咳嗽,喘息和运动能力下降等症状。这些AATD症状的出现和强度在个体之间可能存在很大差异,即使是那些具有相同基因突变的个体。虽然有些人可能没有症状,但其他人可能面临相当大的健康挑战。
Timely detection of AATD is crucial for preventing the progression of the disease, enabling prompt intervention, and optimizing treatment management..
及时检测AATD对于预防疾病进展,及时干预和优化治疗管理至关重要。。
The diagnosis of AATD typically involves blood tests to measure AAT levels and genetic testing to identify specific mutations. AATD is frequently misdiagnosed or delayed in diagnosis as other conditions such as COPD, emphysema, chronic bronchitis, or AAT-associated liver disease, given the common respiratory and hepatitis symptoms they share.
AATD的诊断通常涉及血液检测以测量AAT水平和基因检测以鉴定特定突变。AATD与其他疾病(如COPD,肺气肿,慢性支气管炎或AAT相关肝病)一样,由于其常见的呼吸道和肝炎症状,常被误诊或延迟诊断。
Therefore, a differential diagnosis may pinpoint particular markers in blood or other biological samples associated with AATD..
因此,鉴别诊断可以确定血液或其他与AATD相关的生物样本中的特定标记。。
Alpha-1 Antitrypsin Deficiency Epidemiology Segmentation
α-1抗胰蛋白酶缺乏症流行病学细分
The AATD epidemiology section provides insights into the historical and current AATD patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders.
AATD流行病学部分提供了对历史和当前AATD患者库的见解,并预测了7MM的趋势。通过探索大量研究和关键意见领袖的观点,它有助于识别当前和预测患者趋势的原因。
The AATD market report proffers epidemiological analysis for the study period 2019–2032 in the 7MM segmented into:
AATD市场报告提供了2019-2032年研究期间7MM的流行病学分析,分为:
Total AATD Prevalent Cases
AATD流行病例总数
AATD Genotype-specific Prevalent Cases
AATD基因型特异性流行病例
AATD Comorbidity-associated Prevalent Cases
AATD合并症相关流行病例
Alpha-1 Antitrypsin Deficiency Treatment Market
α-1抗胰蛋白酶缺乏症治疗市场
The existing AATD treatment landscape lacks definitive and disease-altering remedies. Symptomatic relief and the management of lung-related complications such as COPD or emphysema often rely on various off-label drugs, including bronchodilators, corticosteroids, and antibiotics. Augmentation therapy, also known as replacement therapy, is an approved approach for addressing AAT-related lung disease.
现有的AATD治疗方案缺乏明确的和改变疾病的药物。症状缓解和肺部相关并发症(如COPD或肺气肿)的管理通常依赖于各种非标签药物,包括支气管扩张剂,皮质类固醇和抗生素。增强疗法,也称为替代疗法,是一种经批准的治疗AAT相关肺部疾病的方法。
In the severe stages of the ailment, surgical interventions like lung volume reduction surgery and bullectomy are recommended. For individuals with highly advanced emphysema, lung transplant surgery may be necessary, entailing the removal of a damaged lung and its replacement with a healthy one; however, this procedure comes with significant risks, including the potential for infection and rejection..
在疾病的严重阶段,建议进行肺减容手术和大疱切除术等手术干预。对于高度晚期肺气肿的患者,可能需要进行肺移植手术,需要切除受损的肺并用健康的肺替代;然而,这个过程带来了巨大的风险,包括感染和排斥的可能性。。
IV replacement therapy, primarily employed in the treatment of lung disorders, employs AAT protein sourced from the blood plasma of healthy human donors. This approach is applied to enhance alpha-1 levels in individuals diagnosed with emphysema, to elevate the concentration of alpha-1 protein in the lungs.
主要用于治疗肺部疾病的IV替代疗法使用来自健康人类供体血浆的AAT蛋白。这种方法用于提高被诊断患有肺气肿的个体的α-1水平,以提高肺部α-1蛋白的浓度。
The objective is to safeguard the lungs from the harmful impacts of necrotizing panniculitis (NP). The therapy involves a weekly IV infusion and can be administered on a lifelong basis until alternative treatments become accessible..
目的是保护肺部免受坏死性脂膜炎(NP)的有害影响。该疗法涉及每周静脉输注,并且可以终身服用,直到可以获得替代疗法。。
FDA-approved augmentation therapies available for purchase in the US market include PROLASTIN-C by Grifols, ARALAST by Takeda, ZEMAIRA by CSL Behring, and GLASSIA by Kamada. PROLASTIN, introduced in 1988, has maintained a commendable safety record. Subsequently, ARALAST and ZEMAIRA entered the market in 2003, followed by GLASSIA in 2010.
美国市场上可购买的FDA批准的增强疗法包括Grifols的PROLASTIN-C,武田的ARALAST,CSL Behring的ZEMAIRA和Kamada的GLASSIA。1988年推出的PROLASTIN保持着值得称道的安全记录。随后,ARALAST和ZEMAIRA于2003年进入市场,GLASSIA于2010年进入市场。
Each of these therapies received approval based on their safety profiles and their ability to augment alpha-1 levels in the blood and lungs, comparable to PROLASTIN..
这些疗法中的每一种都基于其安全性以及与催乳素相当的增加血液和肺部α-1水平的能力而获得批准。。
To know more about AATD treatment guidelines, visit @ Alpha-1 Antitrypsin Deficiency Management
要了解更多有关AATD治疗指南的信息,请访问@Alpha-1抗胰蛋白酶缺乏症管理
Alpha-1 Antitrypsin Deficiency Pipeline Therapies and Key Companies
α-1抗胰蛋白酶缺乏症管道疗法和关键公司
Inhaled Alpha 1-Antitrypsin (AAT): Kamada Pharmaceuticals
吸入α1-抗胰蛋白酶(AAT):Kamada Pharmaceuticals
Fazirsiran (ARO-AAT/TAK-999): Arrowhead Pharmaceuticals/Takeda
Fazirsiran(ARO-AAT/TAK-999):箭头制药/武田
Alvelestat (MPH-966): Mereo BioPharma/AstraZeneca
Alvelestat(MPH-966):Mereo BioPharma/阿斯利康
VX-864: Vertex Pharmaceuticals
VX-864:Vertex制药
INBRX-101: Inhibrx
INBRX-101:抑制
Belcesiran (DCR-A1AT): Novo Nordisk
比利时(DCR-A1AT):诺和诺德
Learn more about the FDA-approved drugs for AATD @ Drugs for Alpha-1 Antitrypsin Deficiency Treatment
了解更多有关FDA批准用于AATD@α-1抗胰蛋白酶缺乏症治疗药物的药物
Alpha-1 Antitrypsin Deficiency Market Dynamics
α-1抗胰蛋白酶缺乏症市场动态
The dynamics of the alpha-1 antitrypsin deficiency market are expected to change in the coming years. Ongoing research and an increased understanding of diseases have resulted in the identification of therapies featuring effective and convenient routes of administration, such as subcutaneous, inhalation, and oral methods, with the potential to enhance the quality of life for patients.
预计未来几年,α-1抗胰蛋白酶缺乏症市场的动态将发生变化。正在进行的研究和对疾病的更多了解导致了以有效和方便的给药途径为特征的疗法的鉴定,例如皮下,吸入和口服方法,具有提高患者生活质量的潜力。
Active patient registries and supportive organizations play a crucial role in providing information, advocacy, and assistance to individuals with alpha-1 antitrypsin deficiency. Meanwhile, exploration of novel approaches like RNA editing and gene therapy in several preclinical and early-phase assets is underway, offering promising avenues for curative therapies.
积极的患者登记处和支持组织在为α-1抗胰蛋白酶缺乏症患者提供信息,宣传和帮助方面发挥着至关重要的作用。同时,正在几个临床前和早期资产中探索RNA编辑和基因治疗等新方法,为治疗提供了有希望的途径。
Despite the absence of approved therapies for liver disease associated with alpha-1 antitrypsin deficiency, this unmet need presents a strategic opportunity for pharmaceutical players to seize a first-mover advantage in the untapped AATD market..
尽管缺乏针对与α-1抗胰蛋白酶缺乏症相关的肝病的批准疗法,但这种未得到满足的需求为制药企业提供了一个战略机会,可以在尚未开发的AATD市场中占据先发优势。。
Furthermore, many potential therapies are being investigated for the treatment of AATD, and it is safe to predict that the treatment space will significantly impact the AATD market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the AATD market in the 7MM..
此外,正在研究许多治疗AATD的潜在疗法,可以安全地预测,在预测期间,治疗空间将对AATD市场产生重大影响。此外,预计将引入疗效提高的新兴疗法和诊断率的进一步提高,预计将推动AATD市场在7MM的增长。。
However several factors may impede the growth of the alpha-1 antitrypsin deficiency market. The majority of AATD cases remain undiagnosed, with an average delay of over 5 years until diagnosis. Unfortunately, there is no curative treatment for this condition, compelling patients to depend on long-term medication or undergo transplant surgeries in severe cases.
然而,有几个因素可能会阻碍α-1抗胰蛋白酶缺乏症市场的增长。大多数AATD病例仍未确诊,平均延迟5年以上才能确诊。不幸的是,这种情况没有治愈的方法,迫使患者依赖长期药物治疗或在严重情况下接受移植手术。
However, transplants pose risks of rejection and are associated with mortality or morbidity. The rarity and underdiagnosis of the disease create challenges in enrolling a sufficient number of patients for clinical trials, while undefined outcome measures further hinder the development of these trials.
然而,移植会带来排斥风险,并与死亡率或发病率有关。该疾病的罕见性和诊断不足给招募足够数量的患者进行临床试验带来了挑战,而不确定的结局指标进一步阻碍了这些试验的发展。
Additionally, AATD is frequently misdiagnosed due to its similarities with other conditions such as COPD, asthma, and emphysema..
此外,AATD由于与COPD,哮喘和肺气肿等其他疾病的相似性而经常被误诊。。
Moreover, AATD treatment poses a significant economic burden and disrupts patients' overall well-being and QOL. Furthermore, the AATD market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists.
此外,AATD治疗带来了巨大的经济负担,并破坏了患者的整体幸福感和生活质量。此外,AATD市场的增长可能会被新兴疗法的失败和停止、无法负担的定价、市场准入和报销问题以及医疗保健专家的短缺所抵消。
In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the AATD market growth..
此外,未确诊、未报告的病例以及对该疾病的不了解也可能影响AATD市场的增长。。
Alpha-1 Antitrypsin Deficiency Market Report Metrics
Alpha-1抗胰蛋白酶缺乏症市场报告指标
Details
详细信息
Study Period
研究期间
2019–2032
2019–2032
Coverage
覆盖范围
7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].
7MM[美国、欧盟4国(德国、法国、意大利和西班牙)以及英国和日本]。
Alpha-1 Antitrypsin Deficiency Market Size in 2022
2022年Alpha-1抗胰蛋白酶缺乏症的市场规模
USD 1.1 Billion
11亿美元
Key Alpha-1 Antitrypsin Deficiency Companies
关键的α-1抗胰蛋白酶缺乏症公司
Kamada Pharmaceuticals, Arrowhead Pharmaceuticals, Takeda, Mereo BioPharma, AstraZeneca, Vertex Pharmaceuticals, Inhibrx, Novo Nordisk, and others
Kamada Pharmaceuticals、Arrowhead Pharmacecals、Takeda、Mereo BioPharma、AstraZeneca、顶点制药、Inhibrx、Novo Nordisk等
Key Pipeline Alpha-1 Antitrypsin Deficiency Therapies
关键管道α-1抗胰蛋白酶缺乏疗法
PC-61815, Omecamtiv Mecarbil, AZD4831, Rexlemestrocel-L (Revascor), Finerenone (Kerendia), Semaglutide 2.4 mg, Sotagliflozin, Furoscix (furosemide injection), HNO (Nitroxyl) Donor (BMS-986231), Lenrispodun (ITI - 214), Levosimendan, CardiAMPCell Therapy, Tirzepatide, and others
PC-61815、Omecamtiv Mecarbil、AZD4831、Rexremestercel-L(Revascor)、Finerenone(Kerendia)、Semagudide 2.4 mg、Sotagliflozin、Furoscix(呋塞米注射液)、HNO(Nitroxyl)供体(BMS-986231)、Lenresponserdun(ITI-214)、Levosimendan、CardiaMCell Therapy、Tirzepatide等
Scope of the Alpha-1 Antitrypsin Deficiency Market Report
α-1抗胰蛋白酶缺乏症市场报告的范围
Therapeutic Assessment: Alpha-1 Antitrypsin Deficiency current marketed and emerging therapies
治疗评估:α-1抗胰蛋白酶缺乏症目前市场上和新兴的疗法
Alpha-1 Antitrypsin Deficiency Market Dynamics: Key Market Forecast Assumptions of Emerging Alpha-1 Antitrypsin Deficiency Drugs and Market Outlook
α-1抗胰蛋白酶缺乏症市场动态:新兴α-1抗胰蛋白酶缺乏症药物的关键市场预测假设和市场前景
Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
竞争情报分析:SWOT分析与市场进入策略
Unmet Needs, KOL's views, Analyst's views, Alpha-1 Antitrypsin Deficiency Market Access and Reimbursement
未满足的需求,KOL的观点,分析师的观点,α-1抗胰蛋白酶缺乏症的市场准入和报销
Discover more about AATD drugs in development @ Alpha-1 Antitrypsin Deficiency Clinical Trials
了解更多有关AATD药物开发@α-1抗胰蛋白酶缺乏症临床试验的信息
Table of Contents
目录
1.
1.
Alpha-1 Antitrypsin Deficiency Key Insights
α-1抗胰蛋白酶缺乏症的关键见解
2.
2.
Alpha-1 Antitrypsin Deficiency Report Introduction
α-1抗胰蛋白酶缺乏症报告简介
3.
3.
Alpha-1 Antitrypsin Deficiency Overview at a Glance
α-1抗胰蛋白酶缺乏症概述
4.
4.
Alpha-1 Antitrypsin Deficiency Executive Summary
α-1抗胰蛋白酶缺乏症执行摘要
5
5
Alpha-1 Antitrypsin Deficiency Key Events
α-1抗胰蛋白酶缺乏症关键事件
6
6
Epidemiology and Market Forecast Methodology
流行病学和市场预测方法
6.
6.
Disease Background and Overview
疾病背景和概述
7.
7.
Alpha-1 Antitrypsin Deficiency Treatment and Management
α-1抗胰蛋白酶缺乏症的治疗和管理
8.
8.
Alpha-1 Antitrypsin Deficiency Guidelines
α-1抗胰蛋白酶缺乏症指南
9.
9.
Alpha-1 Antitrypsin Deficiency Epidemiology and Patient Population
α-1抗胰蛋白酶缺乏症流行病学和患者人群
10.
10.
Patient Journey
患者旅程
11.
11.
Key Endpoints in Alpha-1 Antitrypsin Deficiency
α-1抗胰蛋白酶缺乏症的关键终点
12.
12.
Alpha-1 Antitrypsin Deficiency Marketed Drugs
α-1抗胰蛋白酶缺乏症市售药物
13.
13.
Alpha-1 Antitrypsin Deficiency Emerging Drugs
α-1抗胰蛋白酶缺乏症新兴药物
14.
14.
7MM Alpha-1 Antitrypsin Deficiency Market Analysis
7MMα-1抗胰蛋白酶缺乏症市场分析
15.
15.
Market Access and Reimbursement
市场准入和报销
16.
16.
KOL Views
KOL Views
17.
17.
Unmet Needs
未满足的需求
18.
18.
SWOT Analysis
SWOT分析
19.
19.
Appendix
附录
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DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve..
DelveInsight是一家专注于生命科学的领先商业顾问和市场研究公司。它通过提供全面的端到端解决方案来提高制药公司的绩效,从而为制药公司提供支持。通过我们基于订阅的平台PharmDelve,轻松访问所有医疗保健和制药市场研究报告。。
Contact UsShruti Thakur [email protected] +91-9650213330www.delveinsight.com
Contact UsShruti Thakur [email protected] +91-9650213330www.delveinsight.com
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SOURCE DelveInsight Business Research, LLP
来源:DelveInsight Business Research,LLP