Amgen granted orphan status for Acute Myeloid Leukemia treatment
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Amicus treatment of CLN6 Batten Disease granted FDA orphan status
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Amgen treatment of aplastic anemia granted FDA orphan designation
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Tetra Bio Pharma treatment of hepatocellular carcinoma granted FDA orphan status
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Amicus treatment of CLN3 Batten disease granted FDA orphan status
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BeiGene treatment of hepatocellular carcinoma granted orphan status
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Constellation granted orphan status for treatment of myelofibrosis
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AbbVie treatment of myelofibrosis granted FDA orphan status
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ProQR Therapeutics treatment of retinal dystrophy in P23H granted orphan status
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Amgen treatment of multiple myeloma granted FDA orphan designation
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Ipsen treatment of pancreatic cancer granted orphan status
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Sangamo granted orphan status for Fabry disease treatment
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BioMarin treatment of phenylketonuria granted FDA orphan status
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Incyte granted orphan status for lymphoma treatment
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Verastem granted orphan status for T-cell lymphoma treatment
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Avrobio treatment of Gaucher disease granted orphan status
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Incyte treatment of follicular lymphoma granted orphan status
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Rhythm Pharmaceuticals treatment of Bardet-Biedl syndrome granted orphan status
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GlaxoSmithKline granted orphan status for biliary cholangitis treatment
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Precision BioSciences treatment of Mantle Cell Lymphoma granted orphan status
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Gossamer Bio granted orphan status for pancreatic cancer treatment
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