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Friedreich Ataxia Management: Introduction

  • Friedreich ataxia is a rare inherited disease leading to progressive nervous system damage and issues in movement. The disease begins in childhood resulting into the impairment of muscle coordination that might get worse over time. It is caused by mutation in a gene labeled FXN. This gene carries the genetic code for a protein ‘frataxin’.
  • Friedreich ataxia results in degeneration of nerve fibers in the spinal cord and peripheral nerves, which later become thinner. The primary function of peripheral nerves is to carry messages or information from the brain to the body and subsequently, from the body back to the brain. These messages are received by the muscles, which signals them to generate movement.
  • Various parts of the brain play an important role in providing signals to the body, one of which is the cerebellum. Cerebellum is a part of the brain that coordinates movement and balance. In case of Friedreich ataxia, cerebellum denegation is also observed to some extent, leading to impaired sensory functions.
  • Currently, there is no treatment for Friedreich ataxia; however, numerous drugs are in the pipeline.

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Key Drivers and Restraints of Global Friedreich Ataxia Management Market

  • The global Friedreich ataxia management market is primarily driven by promising pipeline drugs targeting treatment for Friedreich ataxia, rise in awareness rare diseases, increase in geriatric population, and growing application of precision medicine in neurodegenerative diseases
  • Rise in research & development programs is also projected to boost the market during the forecast period

Hospital Pharmacies segment to account for major share of global Friedreich ataxia management market

  • In terms of distribution channel, the global Friedreich ataxia management market can be classified into hospital pharmacies, retail pharmacies, and online pharmacies
  • The hospital pharmacies segment is anticipated to dominate the global market during the forecast period, due to large inventory maintained by hospitals and availability of essential drugs at hospital pharmacies

North America to dominate global Friedreich ataxia management market

  • In terms of region, the global Friedreich ataxia management market can be segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America is projected to dominate the global Friedreich ataxia management market, followed by Europe.
  • In Europe, increase in number of drugs in clinical trials and rise in research & development expenditure for medication treating neurological conditions is projected to drive the market. In October 2019, Agilis Biotherapeutics received orphan product designation approval for its gene therapy treatment candidate for the treatment of Friedreich Ataxia in Europe. Numerous companies having drugs in pipeline have received positive results in clinical trials. In November 2019, Reata Pharmaceuticals registered positive results for its trial of a drug for Friedreich’s ataxia (FA). The trail included patients from the UCLH Ataxia Centre for the UK.

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Key Players Operating in Global Friedreich Ataxia Management Market

The global Friedreich ataxia management market is highly fragmented, with a large number of domestic players accounting for major market share. Key players operating in the global Friedreich ataxia management market are:

  • Reata Pharmaceuticals, Inc.
  • Agilis Biotherapeutics
  • Biovista
  • Adverum Biotechnologies Inc.
  • Catabasis Pharmaceuticals Inc.
  • RegenxBio
  • Pfizer
  • ProQR Therapeutics NV
  • RaNA Therapeutics Inc.
  • Agios Pharmaceuticals Inc.

Global Friedreich Ataxia Management Market: Research Scope

Global Friedreich Ataxia Management Market, by Form

  • ADVM-063
  • BHV-4157
  • BVA-202

Global Friedreich Ataxia Management Market, by Distribution Channel

  • Hospital Pharmacies
  • Online Pharmacies
  • Retail Pharmacies

This study by TMR is all-encompassing framework of the dynamics of the market. It mainly comprises critical assessment of consumers' or customers' journeys, current and emerging avenues, and strategic framework to enable CXOs take effective decisions.

Our key underpinning is the 4-Quadrant Framework EIRS that offers detailed visualization of four elements:

  • Customer Experience Maps
  • Insights and Tools based on data-driven research
  • Actionable Results to meet all the business priorities
  • Strategic Frameworks to boost the growth journey

The study strives to evaluate the current and future growth prospects, untapped avenues, factors shaping their revenue potential, and demand and consumption patterns in the global market by breaking it into region-wise assessment.

The following regional segments are covered comprehensively:

  • North America
  • Asia Pacific
  • Europe
  • Latin America
  • The Middle East and Africa

The EIRS quadrant framework in the report sums up our wide spectrum of data-driven research and advisory for CXOs to help them make better decisions for their businesses and stay as leaders.

Below is a snapshot of these quadrants.

1. Customer Experience Map

The study offers an in-depth assessment of various customers’ journeys pertinent to the market and its segments. It offers various customer impressions about the products and service use. The analysis takes a closer look at their pain points and fears across various customer touchpoints. The consultation and business intelligence solutions will help interested stakeholders, including CXOs, define customer experience maps tailored to their needs. This will help them aim at boosting customer engagement with their brands.

2. Insights and Tools

The various insights in the study are based on elaborate cycles of primary and secondary research the analysts engage with during the course of research. The analysts and expert advisors at TMR adopt industry-wide, quantitative customer insights tools and market projection methodologies to arrive at results, which makes them reliable. The study not just offers estimations and projections, but also an uncluttered evaluation of these figures on the market dynamics. These insights merge data-driven research framework with qualitative consultations for business owners, CXOs, policy makers, and investors. The insights will also help their customers overcome their fears.

3. Actionable Results

The findings presented in this study by TMR are an indispensable guide for meeting all business priorities, including mission-critical ones. The results when implemented have shown tangible benefits to business stakeholders and industry entities to boost their performance. The results are tailored to fit the individual strategic framework. The study also illustrates some of the recent case studies on solving various problems by companies they faced in their consolidation journey.

4. Strategic Frameworks

The study equips businesses and anyone interested in the market to frame broad strategic frameworks. This has become more important than ever, given the current uncertainty due to COVID-19. The study deliberates on consultations to overcome various such past disruptions and foresees new ones to boost the preparedness. The frameworks help businesses plan their strategic alignments for recovery from such disruptive trends. Further, analysts at TMR helps you break down the complex scenario and bring resiliency in uncertain times.

The report sheds light on various aspects and answers pertinent questions on the market. Some of the important ones are:

1. What can be the best investment choices for venturing into new product and service lines?

2. What value propositions should businesses aim at while making new research and development funding?

3. Which regulations will be most helpful for stakeholders to boost their supply chain network?

4. Which regions might see the demand maturing in certain segments in near future?

5. What are the some of the best cost optimization strategies with vendors that some well-entrenched players have gained success with?

6. Which are the key perspectives that the C-suite are leveraging to move businesses to new growth trajectory?

7. Which government regulations might challenge the status of key regional markets?

8. How will the emerging political and economic scenario affect opportunities in key growth areas?

9. What are some of the value-grab opportunities in various segments?

10. What will be the barrier to entry for new players in the market?

Note: Although care has been taken to maintain the highest levels of accuracy in TMR’s reports, recent market/vendor-specific changes may take time to reflect in the analysis.

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Friedreich Ataxia Management Market

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