Capella

We presented additional pre-clinical data further elucidating fitusiran’s novel mechanism of action at the International Society of Thrombosis and Haemostasis (ISTH) Congress, held July 8 – 13, 2017 in Berlin, Germany. Presentation on the development of a PK-PD model of fitusiran Presentation on measurement of thrombin generation...

We presented pre-clinical data with ALN-F12 at the International Society of Thrombosis and Haemostasis (ISTH) Congress, held July 8 – 13, 2017 in Berlin, Germany. Data showed that reduction of hepatic factor XII (FXII) expression in mice by ALN-F12 inhibits thrombosis without increasing bleeding risk. We believe that reduction of plasma FXII by ALN-F12 represents a promising approach for the prophylactic treatment of thrombosis. View the presentation

We reported new positive results from our ongoing Phase 2 open-label extension (OLE) study with fitusiran in patients with hemophilia A and B, with or without inhibitors, at the International Society of Thrombosis and Haemostasis (ISTH) Congress, held July 8 – 13, 2017 in Berlin, Germany. Read our press release View the complete Phase 2 OLE data presentation

We reported interim results from our ongoing Phase 1 study with givosiran at the 2017 International Congress on Porphyrins and Porphyrias (ICPP), held June 25 – 28, 2017 in Bordeaux, France. Data presented were from the first three unblinded cohorts from Part C, in patients with acute intermittent porphyria (AIP) that experience recurrent attacks and initial data from the open-label extension (OLE) study. Read our press release View the complete Phase 1 interim and OLE data presentation View the results from the EXPLORE natural history study View the poster on healthcare utilization and costs View the poster on disease burden in patients with AIP and recurrent attacks

We presented data evaluating the measurement properties of the Rasch-built Overall Disability Scale (R-ODS) in patients with symptomatic hereditary ATTR (hATTR) amyloidosis with polyneuropathy. R-ODS is a 24-item patient-reported outcome instrument that assesses activity and social participation limitation. R-ODS assessments were collected from two trials investigating patisiran. These data were presented at the International Society for Pharmacoeconomics and Outcomes Research annual meeting in Boston, Massachusetts. [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ISPOR-2017_R-ODS_052417.pdf" type="(1.1 MB PDF)"]View the poster[/spotlight-link]

We reported final 24-month results from the subgroup of hereditary ATTR (hATTR) amyloidosis patients with cardiac involvement in the Phase 2 open-label extension (OLE) study of patisiran at the European Society of Cardiology Heart Failure (ESC-HF) Congress in Paris, France. [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ESC-HF-2017_P2-OLE-Cardiac_04292017.pdf" type="(432 KB PDF)"]View the presentation[/spotlight-link]

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