Capella

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]

We reported final 24-month results from our Phase 2 open-label extension (OLE) study of patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary ATTR (hATTR) amyloidosis. These data were presented at the American Academy of Neurology (AAN) 2017 Annual Meeting in Boston, Massachusetts. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=1022960" type=" "]Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/AAN-2017_Patisiran_Ph-2-OLE_Final-24mos_042617.pdf" type="(940 KB PDF)"]View the presentation[/spotlight-link]

We presented results from a survey evaluating the utility and user experience of an independent genetic testing and counseling service for hereditary ATTR (hATTR) amyloidosis that is supported by Alnylam – Alnylam Act – at the American College of Medical Genetics and Genomics (ACMG) Annual...

Alnylam and The Medicines Company reported final results from the ORION-1 Phase 2 study of inclisiran, an investigational RNAi therapeutic targeting PCSK9 for the treatment of hypercholesterolemia, at the American College of Cardiology (ACC) Scientific Session 2017 in Washington DC. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=1017881" type=" "]Read the press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ACC_Ray-401-18_ORION-1_031717.pdf" type="(380 KB PDF)"]View the presentation[/spotlight-link]

Alnylam is sponsoring free, third-party genetic testing and counseling for individuals who may carry a gene mutation known to be associated with hereditary ATTR (hATTR) amyloidosis. The Alnylam Act™ (formerly known as Alnylam Assist) program was created to potentially increase diagnosis rates and to provide...

We reported new results from an exploratory analysis of our Phase 1 study with fitusiran, an investigational RNAi therapeutic, in patients with hemophilia A or B with or without inhibitors at the 10th Annual Congress of the European Association of Haemophilia and Allied Disorders (EAHAD) held February 1 – 3, 2017 in Paris, France. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=1010213" type=" "]Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/EAHAD-2017_Management-of-Bleed-Events.pdf" type="(450 KB PDF)"]View the bleed management presentation[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/EAHAD-2017_Stability.pdf" type="(690 KB PDF)"]View the stability study presentation[/spotlight-link]

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