22 May, 2020 New Results for Patisiran and Givosiran at EAN

We presented new long-term results from the global Open-Label Extension (OLE) study of ONPATTRO^® (patisiran) at the European Academy of Neurology (EAN) Virtual Congress, held May 23-26, 2020. In addition, results were presented from a Phase 3b trial evaluating treatment with patisiran in patients with hATTR amyloidosis with disease progression after receiving an orthotopic liver transplant (post-OLT). A post-hoc analysis of the ENVISION Phase 3 study of GIVLAARI^® (givosiran) was also presented.

Read the press release

View the patisiran global OLE poster

View the patisiran post-OLT poster

View the ENVISION post-hoc analysis

View the presentation on disease burden and healthcare utilization among patients with acute intermittent porphyria

(more…)

14 Apr, 2020 New Clinical Results for Givosiran at EASL

An abstract describing new 12-month interim data from the ongoing ENVISION open-label extension (OLE) study of givosiran in patients with acute hepatic porphyria (AHP) has been published online by the European Association for the Society of the Liver (EASL) on the International Liver Congress™ (ILC 2020) website.  The data will be presented during the ILC 2020 (London, UK) which was rescheduled to August 25-28, 2020 due to the COVID-19 pandemic.

View the ENVISION OLE abstract

(more…)

09 Nov, 2019 New Clinical Results for Lumasiran at ASN

We presented new positive efficacy results from the ongoing Phase 2 open-label extension (OLE) study of lumasiran, an investigational subcutaneously administered RNAi therapeutic in development for the treatment of primary hyperoxaluria type 1 (PH1), at the American Society of Nephrology (ASN) 2019 Annual Meeting, held November 5-10 in Washington, DC.

Read our press release

View the Phase 2 OLE poster

(more…)

07 Nov, 2019 EXPLORE Natural History Study Data Published in “Hepatology”

We published results from the EXPLORE natural history study of more than 100 acute hepatic porphyria (AHP) patients with recurrent attacks in Hepatology in a paper titled, “EXPLORE: A Prospective, Multinational, Natural History Study of Patients with Acute Hepatic Porphyria with Recurrent Attacks.”

Read the paper in Hepatology

(more…)

20 Oct, 2019 Results from Pediatric Cohort of Phase 1/2 Study of Lumasiran in Patients with Primary Hyperoxaluria Type 1 (PH1) and Insights from a Survey of Physician Experiences with PH1

We presented results on the pediatric cohort of patients (N=16; age range 6-17) from the Phase 1/2 study of lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of primary hyperoxaluria type 1 (PH1), at the International Pediatric Nephrology Association (IPNA) 2019 Annual Meeting, held October 17-21 in Venice, Italy.

Frishberg et al. – Phase 1/2 Study of Lumasiran, Investigational RNAi Therapeutic, in Patients with Primary Hyperoxaluria Type 1

Danese et al. – Understanding the Burden of Primary Hyperoxaluria Type 1 (PH1): A Survey of Physician Experiences with PH1

(more…)

13 Sep, 2019 New Evidence Demonstrating Significant Association of V122I TTR Gene Mutation with Clinical Diagnosis of Polyneuropathy

We presented new results from an analysis of the UK Biobank demonstrating a significant association of the V122I mutation, a highly prevalent mutation in the transthyretin (TTR) gene, with a clinical diagnosis of polyneuropathy. These results were presented at the Heart Failure Society of America (HFSA) 23^rd Annual Scientific Meeting, held September 13-16 in Philadelphia, PA.

Read our press release

Parker et al. – “The V122I Mutation in Hereditary Transthyretin-Mediated Amyloidosis is Significantly Associated with Polyneuropathy”

(more…)