Study to assess a diagnostic protocol for individuals with unexplained splenomegaly and thrombocytopenia

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Sanofi Genzyme is currently looking for sites to participate in the feasibility for an upcoming Sanofi Genzyme study to assess a diagnostic protocol for individuals with unexplained splenomegaly and thrombocytopenia. This study will determine the prevalence of Gaucher Disease and Acid Sphingomyelinase Deficiency (Niemann-Pick A and B) among individuals with splenomegaly and thrombocytopenia who meet additional inclusion… read more //

ENVISION Phase 3 Study

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Evaluating the effect of givosiran (ALN-AS1) on the rate of porphyria attacks in patients with Acute Hepatic Porphyrias (AHP). Trial Status: Enrolling Interested in learning more? Please email us at [email protected] For more info: ClinicalTrials.gov – NCT03338816