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’.
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- 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
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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.
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