Amicus Therapeutics rare disease drug effective in trial

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Amicus Therapeutics rare disease drug effective in trial

Amicus Therapeutics Inc said its experimental drug significantly reduced the abnormal accumulation of fat in body cells related to a rare genetic disorder that could lead to heart attack, stroke and kidney failure. The late-stage trial evaluated the drug, migalastat, as a form of monotherapy for certain Fabry disease patients after 12 months of treatment. "It may be that (the drug) needs more time to work, as shown by today's results," Janney Montgomery Scott analyst Kimberly Lee said, adding that the European trial data would provide further evidence of whether the drug needed a longer duration to be effective.

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