Japan AADC Deficiency Market Report 2023 to 2030

Japan AADC Deficiency Market Analysis

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Japan AADC Deficiency Market is projected to grow from $xx Mn in 2023 to $xx Mn by 2030, registering a CAGR of xx% during the forecast period of 2023-30. The growing awareness of rare diseases like AADC deficiency among the general public and medical community is one of the major factors influencing market expansion. More cases are being diagnosed as the illness gains recognition, which results in early treatment and better patient outcomes. As medical professionals become more familiarized with the illness and its symptoms, it is expected that this tendency will continue.Major global players in AADC Deficiency Market are Lysogene, Retrophin, Ultragenyx Pharmaceutical, PTC Therapeutics, Azevan Pharmaceuticals, Neurocrine Biosciences, Adrenomed AG, Renova Therapeutics, Titan Pharmaceuticals, Voyager Therapeutics.

ID: IN10JPRD070 CATEGORY: Rare Diseases GEOGRAPHY: Japan AUTHOR: Nipun Arora

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Japan AADC Deficiency Market Analysis Summary

Japan AADC Deficiency Market is valued at around $12.1 Mn in 2022 and is projected to reach $20.49 Mn by 2030, exhibiting a CAGR of 6.8% during the forecast period 2023-2030.

A unique genetic condition known as AADC deficiency affects the synthesis of multiple neurotransmitters in the brain. Despite the disorder's extraordinary rarity, those who have it and those who love them could suffer negative effects. Analytically, AADC insufficiency demonstrates a significant area of unmet medical need because there are no proven treatments to address the underlying cause of the ailment. Sometimes it is possible to treat the disease's symptoms, but this is not a cure and does not correct the underlying genetic abnormality. To fully comprehend the fundamental genetic and molecular causes of AADC insufficiency and to create specialized therapies that target these causes, more study is needed.

Gene therapy and enzyme replacement therapy research in the future may lead to treatments that are more efficient than those currently in use. The growing awareness of rare diseases like AADC deficiency among the general public and medical community is one of the major factors influencing market expansion. More cases are being diagnosed as the illness gains recognition, which results in early treatment and better patient outcomes. As medical professionals become more familiarized with the illness and its symptoms, it is expected that this tendency will continue. Major global players in AADC Deficiency Market are Lysogene, Retrophin, Ultragenyx Pharmaceutical, PTC Therapeutics, Azevan Pharmaceuticals, Neurocrine Biosciences, Adrenomed AG, Renova Therapeutics, Titan Pharmaceuticals, Voyager Therapeutics.

Market Dynamics

Market Drivers

The growing public and professional understanding of rare disorders, such as AADC deficiency, is one of the major factors influencing market expansion. The market for AADC deficiency is also expected to grow as a result of research advances. Ongoing research into the genetic and molecular roots of the illness is leading to the development of new and more effective treatments. Gene therapy and enzyme replacement therapy are two exciting areas of research with the potential to develop novel treatments for AADC deficiency. When these medications are developed and made available on the market, there will likely be greater demand for AADC-deficient treatments. The market expansion for AADC insufficiency is also influenced by regulatory incentives. Regulatory organizations like the FDA and EMA support the development of medications to treat unusual illnesses like AADC deficiency. One of these rewards is the orphan drug designation, which can offer financial and legal advantages to businesses creating drugs to treat uncommon diseases. With more options for patients and a growing market, these incentives are anticipated to motivate more businesses to invest in researching and developing treatments for AADC insufficiency.

Developments in Japan AADC Deficiency Market

On Nov 17, 2022: PTC Therapeutics, Inc. (NASDAQ: PTCT) announced that Upstaza™ (eladocagene exuparvovec) was granted authorization by the Medicines and Healthcare Products Regulatory Agency (MHRA) in Great Britain. Upstaza is the first and only approved disease-modifying treatment for aromatic L-amino acid decarboxylase (AADC) deficiency and the first marketed gene therapy directly infused into the brain. It is approved for patients 18 months and older.

Key players

uniQure Ionis Pharmaceuticals Alnylam Pharmaceuticals Catalyst Biosciences Pfizer Sanofi Shire plc BioMarin Pharmaceutical Inc. Orchard Therapeutics plc PTC Therapeutics Inc.

1. Executive Summary
1.1 Disease Overview
1.2 Global Scenario
1.3 Country Overview
1.4 Healthcare Scenario in Country
1.5 Patient Journey
1.6 Health Insurance Coverage in Country
1.7 Active Pharmaceutical Ingredient (API)
1.8 Recent Developments in the Country

2. Market Size and Forecasting
2.1 Epidemiology of Disease
2.2 Market Size (With Excel & Methodology)
2.3 Market Segmentation (Check all Segments in Segmentation Section)

3. Market Dynamics
3.1 Market Drivers
3.2 Market Restraints

4. Competitive Landscape
4.1 Major Market Share

4.2 Key Company Profile (Check all Companies in the Summary Section)

4.2.1 Company
4.2.1.1 Overview
4.2.1.2 Product Applications and Services
4.2.1.3 Recent Developments
4.2.1.4 Partnerships Ecosystem
4.2.1.5 Financials (Based on Availability)

5. Reimbursement Scenario
5.1 Reimbursement Regulation
5.2 Reimbursement Process for Diagnosis
5.3 Reimbursement Process for Treatment

6. Methodology and Scope

Japan AADC Deficiency Market Segmentation

By Treatment:

Gene therapy
Enzyme replacement therapy
Symptomatic treatment
Combination therapy

By Diagnostics:

Genetic testing
Biochemical testing
Enzyme activity testing
Others

By End Users:

Hospitals
Clinics
Specialized treatment centers
Others

Methodology for Database Creation

Our database offers a comprehensive list of healthcare centers, meticulously curated to provide detailed information on a wide range of specialties and services. It includes top-tier hospitals, clinics, and diagnostic facilities across 30 countries and 24 specialties, ensuring users can find the healthcare services they need.

Additionally, we provide a comprehensive list of Key Opinion Leaders (KOLs) based on your requirements. Our curated list captures various crucial aspects of the KOLs, offering more than just general information. Whether you're looking to boost brand awareness, drive engagement, or launch a new product, our extensive list of KOLs ensures you have the right experts by your side. Covering 30 countries and 36 specialties, our database guarantees access to the best KOLs in the healthcare industry, supporting strategic decisions and enhancing your initiatives.

How Do We Get It?

Our database is created and maintained through a combination of secondary and primary research methodologies.

1. Secondary Research

With many years of experience in the healthcare field, we have our own rich proprietary data from various past projects. This historical data serves as the foundation for our database. Our continuous process of gathering data involves:

Analyzing historical proprietary data collected from multiple projects.
Regularly updating our existing data sets with new findings and trends.
Ensuring data consistency and accuracy through rigorous validation processes.

With extensive experience in the field, we have developed a proprietary GenAI-based technology that is uniquely tailored to our organization. This advanced technology enables us to scan a wide array of relevant information sources across the internet. Our data-gathering process includes:

Searching through academic conferences, published research, citations, and social media platforms
Collecting and compiling diverse data to build a comprehensive and detailed database
Continuously updating our database with new information to ensure its relevance and accuracy

2. Primary Research

To complement and validate our secondary data, we engage in primary research through local tie-ups and partnerships. This process involves:

Collaborating with local healthcare providers, hospitals, and clinics to gather real-time data.
Conducting surveys, interviews, and field studies to collect fresh data directly from the source.
Continuously refreshing our database to ensure that the information remains current and reliable.
Validating secondary data through cross-referencing with primary data to ensure accuracy and relevance.

Combining Secondary and Primary Research

By integrating both secondary and primary research methodologies, we ensure that our database is comprehensive, accurate, and up-to-date. The combined process involves:

Merging historical data from secondary research with real-time data from primary research.
Conducting thorough data validation and cleansing to remove inconsistencies and errors.
Organizing data into a structured format that is easily accessible and usable for various applications.
Continuously monitoring and updating the database to reflect the latest developments and trends in the healthcare field.

Through this meticulous process, we create a final database tailored to each region and domain within the healthcare industry. This approach ensures that our clients receive reliable and relevant data, empowering them to make informed decisions and drive innovation in their respective fields.