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Updated Results from Phase 1/2 Study of ALN-CC5 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Updated Results from Phase 1/2 Study of ALN-CC5 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

We reported new results from Part C of our Phase 1/2 clinical study of ALN-CC5, a subcutaneously administered investigational RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases, at the 58th Annual Meeting of the American Society of Hematology (ASH), held December 3 – 6, 2016 in San Diego, California.



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Interim Clinical Results from Fitusiran, an investigational RNAi therapeutic for the Treatment of Hemophilia and Rare Bleeding Disorders

Interim Clinical Results from Fitusiran, an investigational RNAi therapeutic for the Treatment of Hemophilia and Rare Bleeding Disorders

We reported positive interim results from our Phase 1 study of fitusiran in patients with hemophilia with inhibitors as well as from our Phase 2 open-label extension (OLE) study in hemophilia patients without inhibitors at the 58th Annual Meeting of the American Society of Hematology (ASH), held December 3 – 6, 2016 in San Diego, California.





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Initial Evidence of Clinical Activity Shown with Givosiran (ALN-AS1) in Acute Intermittent Porphyria Patients with Recurrent Attacks

Initial Evidence of Clinical Activity Shown with Givosiran (ALN-AS1) in Acute Intermittent Porphyria Patients with Recurrent Attacks

We reported positive initial results from Cohorts 1 and 2 in Part C of our Phase 1 study with givosiran (gi-VOH-si-ran), the International Nonproprietary Name for ALN-AS1, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyrias.  These results were presented at the 58th Annual Meeting of the American Society of Hematology (ASH), held December 3 – 6, 2016 in San Diego, California.



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Alnylam WHO

From Possibility to Patients

In 2006, Craig Mello, Ph.D., and Andrew Fire, Ph.D., received the Nobel Prize for Medicine for the discovery of RNA Interference (RNAi). Alnylam is recognizing the 10th anniversary of this historic achievement and the progress made toward delivering RNAi therapeutics, an entirely new class of innovative medicines to patients. Explore this page to learn more about our story and the scientific journey of RNAi—from possibility, to potential treatment for patients.


Alnylam Pharmaceuticals Supports Greater Boston and Norton Communities with its 2nd Annual Helping Hands Community Service Day

Alnylam Pharmaceuticals Supports Greater Boston and Norton Communities with its 2nd Annual "Helping Hands" Community Service Day

On June 23, over 400 Alnylam employees participated in its second annual company-wide “Helping Hands” Community Service Day to benefit ten local non-profit organizations including: Boston Nature Center, Cradles to Crayons, Emerald Necklace Conservancy, Friends of Alewife, Habitat Education Center, IMEC America, Cupboard of Kindness, Norton Conservation Land, West End House Boys and Girls Club and Windrush Farms.


Behind the Mystery: Rare + Genetic Series - TTR Amyloid

Behind the Mystery: Rare & Genetic Series - TTR Amyloid

Battling a rare medical condition, that's often misdiagnosed.
Imagine having a rare, debilitating disease that's passed down through families. Dr. John Berk, Associate Professor of Medicine at Boston University and Clinical Director of its Amyloidosis Center, joins us to discuss the medical condition called Transthyretin-Mediated Amyloidosis. It's symptoms are similar to other conditions and ATTR patients are very often misdiagnosed. We discuss symptoms, family connections and treatment options, in this Behind the Mystery: Rare & Genetic segment. And meet Dylan Duncan, who's battling the disease. You don't want to miss this.