US Freidreich's Ataxia Market Report 2023 to 2030

US Freidreich's Ataxia Market Analysis

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US Freidreich?s Ataxia 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 - 2030. The market for Freidreich?s Ataxia is growing rapidly as a result of increase in research and developmental activities for diagnosis and treatment of freidreich?s ataxia, collaborations between academic institutions and industry partners and incentives provided in orphan drug designation by regulatory authorities. PTC Therapeutics, Design Therapeutics, Aurobindo Pharma Ltd., Sun Pharmaceutical Industries Ltd., Torrent Pharmaceuticals Ltd., Intas Pharmaceuticals Ltd., Larimar Therapeutics, Inc., Jupiter Neurosciences, Inc., Lexeo Therapeutics, Zydus Lifesciences Ltd., Cipla Limited, GlaxoSmithKline Plc., Reata Pharmaceuticals, Inc., and Retrotope Inc. are the key global market players operating in global freidreich?s Ataxia Market.

ID: IN10USRD086 CATEGORY: Rare Diseases GEOGRAPHY: US AUTHOR: Nipun Arora

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US Freidreich's Ataxia Market Analysis Summary

US Freidreich's Ataxia Market is valued at around $0.33 Bn in 2022 and is projected to reach $0.76 Bn by 2030, exhibiting a CAGR of 11% during the forecast period 2023-2030.

Friedreich's ataxia is a genetic, neurodegenerative movement disorder with symptoms like muscle weakness and impaired speech. It is caused by genetic mutations resulting in a decline in the production and expression of a mitochondrial protein called frataxin, which is involved in regulating iron-sulphur cluster enzymes within cells of the body. Insufficient frataxin levels result in the reduced activity of several mitochondrial enzymes involved in the electron transport chain and reduced ATP (Adenosine triphosphate) production, and inefficient anti-oxidant response. The primary goals of treating Friedreich's ataxia include relieving symptoms and resolving any related consequences. Physical therapy, occupational therapy, speech therapy, and the use of assistive technology are all possible treatment modalities. Research into potential disease-modifying treatments is continuing, including efforts to raise frataxin protein levels and antioxidants. Physiotherapy, rehabilitation programs, physical training, etc. for the management of Friedreich’s ataxia are in high demand and can further develop in future markets.

PTC Therapeutics, Design Therapeutics, Larimar Therapeutics Inc., Jupiter Neurosciences, Inc., Lexeo Therapeutics, Reata Pharmaceuticals Inc and Retrotope Inc. are the main market players operating globally.

Market Dynamics

Market Drivers

Collaborative efforts in research and development – Collaboration between academic institutions, industry partners, and patient advocacy groups for research and development acts as a market growth driver.

Orphan drug designations by regulatory authorities – These designations provide incentives like market exclusivity and financial benefits and this can act as a market growth driver.

Patient advocacy and support – Patient advocacy groups help to increase awareness about the disease, provide support to patients suffering from the disease and thus act as a market growth driver.

Market Restraints

Challenges faced during designing and conducting clinical trials for the development of Freidreich's ataxia treatment because of an insufficient number of Friedreich's ataxia patients, the heterogeneous nature of the disease with a slow and variable progression rate, which makes it difficult to identify reliable outcome measures to monitor the progression of Friedreich's ataxia. This acts as a market growth restraint.

The typical age of onset (the age at which the symptoms of the disease first appear) is between 10 and 15 years. Thus, the diagnosis of the disease takes a long time since its initial stage. This also acts as a market growth restraint

Key players

Reata Pharmaceuticals, Inc. Pfizer Inc. BioMarin Pharmaceutical Inc. Retrotope, Inc. Horizon Therapeutics plc Edison Pharmaceuticals, Inc. Santhera Pharmaceuticals PTC Therapeutics, Inc. Vertex Pharmaceuticals Incorporated Biogen 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

Market Segmentations For US Freidreich’s Ataxia market

By Diagnosis

CT scan
MRI scan
Electromyography
Others

By Treatment type

Medication

Beta blockers
ACE inhibitors
Diuretics
Others

Speech therapy
Occupational therapy
Surgeries
Others

By Route of Administration

Oral
Parenteral
Others

By end users

Hospitals
Diagnostic centres
Clinics
Others

By Distribution Channels:

Hospital pharmacies
Retail pharmacies
Online pharmacies
Direct tender
Others

Methodology for Database Creation

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How Do We Get It?

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

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Analyzing historical proprietary data collected from multiple projects.
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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.
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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.
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