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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.
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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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.