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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.