Capella

We presented updated results from the ongoing Phase 1/2 open-label extension (OLE) study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria (AHP), at The Liver Meeting® 2018 of the American Association for the Study of Liver Diseases (AASLD), held November 9-13, 2018 in San Francisco, CA. Read our press release Anderson et al. – “Phase 1/2 and Open Label Extension Studies of Givosiran, an Investigational RNA Interference (RNAi) Therapeutic, in Patients with Acute Intermittent Porphyria” Bonkovsky et al. – “EXPLORE: A Prospective, Multinational, Natural History Study of Acute Hepatic Porphyrias (AHP) Patients with Recurrent Attacks”

We presented new results from the Phase 1/2 and Phase 2 open-label extension (OLE) studies of lumasiran at the American Society of Nephrology (ASN) 2018 Annual Meeting, held October 23-28, 2018 in San Diego, CA. Read our press release Hulton et al. - "Safety and Efficacy Study of Lumasiran (ALN-GO1), an Investigational RNA Interference (RNAi) Therapeutic, in Patients with Primary Hyperoxaluria Type 1"

We presented new 12-month efficacy and safety data with patisiran from the APOLLO Global Open Label Extension (OLE) Study at the 2018 American Association of Neuromuscular & Electrodiagnostic Medicine Annual Meeting, held October 10-13, 2018 in Washington, DC. Gonzalez-Duarte et al. - "Long-Term Use of Patisiran,...

We presented new research on the diagnostic patterns, clinical experience and healthcare utilization of patients with acute hepatic porphyrias at the American College of Gastroenterology 2018 Annual Scientific Meeting, held October 5-10, 2018 in Phildelphia, PA. Ko et al. - "Real-World Analysis of Symptoms, Diagnostic Patterns, and...

We presented updated positive results from the Phase 1/2 clinical study of lumasiran, an investigational, subcutaneously administered RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of primary hyperoxaluria type 1 (PH1), at the 2018 European Society for Paediatric Nephrology (ESPN) Annual Meeting, held October 3-6, 2018 in Antalya, Turkey. Read our press release View the presentation

Data from exploratory cardiac assessments and post-hoc analyses of safety data in the APOLLO Phase 3 study of patisiran, an RNAi therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults, were published in the journal Circulation. Read the paper in Circulation...

On September 11, 2018, we hosted an online RNAi Roundtable to review the progress with ONPATTRO™ (patisiran) and ALN-TTRsc02, RNAi therapeutics for the treatment of transthyretin-mediated amyloidosis*. Access the replay View the presentation Read the transcript * ONPATTRO is approved in the U.S. for the polyneuropathy of hereditary ATTR (hATTR) amyloidosis in adults, and in the EU for the treatment of hATTR amyloidosis in adults with stage 1 or stage 2 polyneuropathy. **ALN-TTRsc02 is an investigational RNAi therapeutic, in development for the treatment of ATTR amyloidosis. 

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