News Charities unite to oppose NICE rare disease rules MPs to discuss controversial plan in special debate.
News FDA fast tracks Shire clotting disease drug The US regulator has given a Fast Track designation to a Shire pipeline drug for a life-threatening blood clotting disease, hereditary thrombotic thrombocytopenic purpura.
Rare diseases Marathon sells Duchenne drug to PTC PTC says it will make changes to the price after abandoned launch last month.
News Teva files orphan blockbuster hope in tardive dyskinesia SD-809 will be vital for Teva if approved.
News Pharma must slash rare disease drug prices - patient organis... More rare disease drugs must also be approved, says EURORDIS.
News Outrage at Marathon's 'new drug' price, calls for PhRMA to a... Marathon under fire after US price hike.
News Neurogene drops high-dose arm in Rett study after death The patient affected by a serious adverse event in Neurogene's clinical trial of Rett syndrome gene therapy has now died
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