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.
QUESTIONS ANSWERED
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?
PRODUCT DESCRIPTION
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.