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Research & Reports

Searching in Biopharma (1746)

Friedreich's Ataxia | Landscape & Forecast | Disease Landscape & Forecast

Friedreich's Ataxia | Landscape & Forecast | Disease Landscape & Forecast

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Friedreich’s ataxia (FA) is a progressive, autosomal recessive, neurodegenerative disease characterized by slowly advancing ataxia (loss of bodily control). No treatments are currently approved for FA; thus, patients rely on a combination of off-label therapies targeted to specific symptoms, over-the-counter antioxidants, and nonpharmacological interventions to manage their disease. Despite the high unmet need for effective treatments, the late-stage FA pipeline is sparse; the most advanced agents are only in Phase II. However, optimism remains high among thoughtleaders as technological breakthroughs in molecular biology and gene-editing techniques are spurring early-stage development in FA. Given the lack of current therapies for FA, significant commercial opportunity remains for any agent that can alleviate the disease’s symptoms or modify its course.


  • How large is the diagnosed prevalent FA population in the United States and EU5? How will the population change through 2027?
  • What is the current treatment landscape for FA patients, and how will it change in the next ten years?
  • What clinical needs remain unfulfilled?
  • Which emerging therapies do neurologists consider most promising, and which are likely to launch by 2027? What will be the commercial impact of these new FA therapies?


Niche & Rare Disease Landscape & Forecast: comprehensive market intelligence providing world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.

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  • Pub Date: May 2018
  • Author(s): Laura Darnieder; Amy Bradshaw Kaiser, MS
  • Laura Darnieder is a Business Insights Analyst on the Infectious, Niche, and Rare Diseases team at Decision Resources Group, where she is focused on providing market forecasts within the niche biopharmaceutical space. Prior to joining DRG, Laura earned a in Neuroscience from Tufts University Sackler School of Graduate Biomedical Sciences. Her research focused on the role of stress neuropeptides and inhibitory signaling in alcohol dependence and drug addiction. She also holds a , summa cum laude, in Psychobiology and Linguistics from Binghamton University (SUNY).

  • Amy Kaiser joined Decision Resources Group as an associate epidemiologist in 2017. Her focus is on the epidemiology of infectious diseases and niche and rare diseases. She holds an MS in Epidemiology from the University of Massachusetts, Amherst and a BA in International Relations from Mount Holyoke College. Prior to joining Decision Resources Group, she worked as a human health research associate at a environmental consulting firm where her epidemiology research focused on occupational and environmental exposures and associated outcomes.

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