Potential Treatment for Niemann-Pick Disease is Granted Orphan Drug Designation in the EU
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Potential Treatment for Niemann-Pick Disease is Granted Orphan Drug Designation in the EU

According to a story from rdmag.com, the biopharmaceutical company IntraBio Inc., recently announced that its IB1000 series of investigational therapeutic compounds have been granted Orphan Medicinal Drug Designation as a…

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This Company Just Endorsed New Patient Advocacy Guidelines
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This Company Just Endorsed New Patient Advocacy Guidelines

According to a story from globenewswire.com, the biopharmaceutical company Orphazyme has officially endorsed a set of guidelines that outline how rare disease patient advocacy organizations and drug companies should interact…

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Clinical Trial Results Look Promising For Experimental Niemann-Pick Disease Type C Treatment
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Clinical Trial Results Look Promising For Experimental Niemann-Pick Disease Type C Treatment

According to a story from BioSpace, the biopharmaceutical company Orphazyme recently announced the results from its Phase 2/3 clinical trial of its investigational drug arimoclomol. This therapy is in development…

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Phase II Trial for Investigational Gaucher Disease Drug Begins
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Phase II Trial for Investigational Gaucher Disease Drug Begins

According to a story from GlobeNewsWire, the biopharmaceutical company Orphazyme A/S, which is committed to the development of treatments for patients that are living with rare diseases, has recently announced…

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