France 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.
France AADC Deficiency Market is valued at around $5.3 Mn in 2022 and is projected to reach $8.02 Mn by 2030, exhibiting a CAGR of 5.3% 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 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.
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
Shire Alexion Pharmaceuticals BioMarin Pharmaceutical Ionis Pharmaceuticals Pfizer Genzyme Sangamo Therapeutics Magenta Therapeutics Ceregene Syndax Pharmaceuticals1. 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
By Treatment:
By Diagnostics:
By End Users:
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