NIH Completes Collaborative Phase II Study with Santhera's compound SNT-MC17/idebenone in Friedreich's Ataxia (FRDA)
The trial enrolled a total of 48 patients, with 12 patients in each of three dose groups and a placebo group. Data from this trial support the favourable safety profile of the compound. Preliminary analysis of the trial results suggests that SNT-MC17/idebenone has a trend toward dose proportional improvement in neurological parameters in FRDA patients as determined by the International Cooperative Ataxia Rating Scale (ICARS), a semi-quantitative scale based upon 19 test parameters that assess sensory and motor skills.
These new data reinforce Santhera's belief that SNT-MC17/idebenone has the potential to provide a possible treatment for this disease.
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