In a recent study, patients with myelodysplastic syndrome (MDS) who have myelofibrosis (MF) showed a worse prognosis when MF was of greater severity. However, this pattern of severity influencing prognosis appeared negated with allogeneic hematopoietic stem cell transplantation (allo-HSCT). The study findings were reported in the journal Experimental Hematology & Oncology....
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SAN FRANCISCO – AllStripes, a healthcare technology company dedicated to accelerating treatment research for patients with rare diseases, today announced a collaboration with the National Center for Advancing Translational Sciences (NCATS), an institute of the NIH established to transform the translational process so that new treatments and cures for disease...
PARIS, France and CAMBRIDGE, MA, USA – Sanofi and Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced a strategic restructuring of their RNAi therapeutics alliance to streamline and optimize development and commercialization of certain products for the treatment of rare genetic diseases. Specifically: Sanofi will obtain global development and commercialization rights...
CAMBRIDGE, Mass. — Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the U.S. Food and Drug Administration (FDA) approval of the supplemental New Drug Application (sNDA) for its RNAi therapeutic, AMVUTTRA® (vutrisiran), for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to...
MISSISSAUGA, ON – Alnylam Canada ULC is pleased to announce that Health Canada has issued a Notice of Compliance (NOC) authorizing AMVUTTRA® (vutrisiran) for the treatment of cardiomyopathy in adult patients with wild-type or hereditary transthyretin-mediated amyloidosis (wtATTR or hATTR amyloidosis).1 The approval broadens the indication for AMVUTTRA®, which now becomes the first and...
CAMBRIDGE, Mass. – Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that it has started a clinical study of a biannual dosing regimen of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis. The study is being conducted during a randomized treatment extension...
CAMBRIDGE, Mass. – Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today positive early results on clinical outcome measures from the 12-month analysis of ILLUMINATE-A Phase 3 study of OXLUMO® (lumasiran), an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) –...
CAMBRIDGE, Mass. – Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that pivotal trial results from the ILLUMINATE-A Phase 3 study of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – for the treatment of primary hyperoxaluria type 1 (PH1), were published online in...
CAMBRIDGE, Mass. – Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced that pivotal trial results from the ILLUMINATE-A Phase 3 study of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – for the treatment of primary hyperoxaluria type 1...
CAMBRIDGE, Mass. – Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the U.S. Food and Drug Administration (FDA) has accepted the Company’s New Drug Application (NDA) for vutrisiran, an investigational RNAi therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults....