News PTC's muscular dystrophy drug can stay on EU market - regula... But PTC must conduct further trial to confirm Translarna's benefits.
News Biogen/Ionis could launch rare disease drug within weeks Companies prepare for launch of Spinraza after phase 3 success.
News Vtesse's rare disease drug set for UK early access Vtesse drug "promising" treatment for NPC disease, says regulator.
News Moderna jab on trial for cancer-causing syndrome Patients with Lynch syndrome could have a potential treatment on the horizon, as a Moderna mRNA vaccine starts trials at Oxford University.
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