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Orphan designation: for: Treatment of pulmonary arterial hypertension
Date of first decision: 29/05/2019, Positive, Last updated: 09/08/2019 -
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Orphan designation: for: Treatment of acute myeloid leukaemia
Date of first decision: 29/05/2019, Positive, Last updated: 09/08/2019 -
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Orphan designation: for: Treatment of epidermolysis bullosa
Date of first decision: 29/05/2019, Positive, Last updated: 09/08/2019 -
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Orphan designation: for: Treatment of Stargardt's disease
Date of first decision: 29/05/2019, Positive, Last updated: 09/08/2019 -
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Orphan designation: for: Treatment of cystic fibrosis
Date of first decision: 29/05/2019, Positive, Last updated: 09/08/2019 -
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Orphan designation: for: Treatment of Cushing's syndrome
Date of first decision: 29/05/2019, Positive, Last updated: 09/08/2019 -
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Orphan designation: for: Treatment of citrullinaemia type 1
Date of first decision: 29/05/2019, Positive, Last updated: 12/08/2019 -
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Orphan designation: for: Treatment of carbamoyl-phosphate synthase-1 deficiency
Date of first decision: 29/05/2019, Positive, Last updated: 12/08/2019 -
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Orphan designation: for: Treatment of lymphoplasmacytic lymphoma
Date of first decision: 29/05/2019, Positive, Last updated: 12/08/2019 -
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Orphan designation: for: Treatment of epidermolysis bullosa
Date of first decision: 29/05/2019, Positive, Last updated: 19/08/2019