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hemophilia

Alnylam Pharmaceuticals this week presented preclinical data from its hemophilia and bleeding disorder program, showing that its subcutaneously administered drug candidate ALN-AT3 could trigger potent, dose-dependent, durable target knockdown in non-human primates.

This article has been updated from a version posted Nov. 13 to correct the name of the National Hemophilia Foundation.

NEW YORK (GenomeWeb News) – Two patient advocacy groups, Biogen Idec, and a medical center have teamed up to offer free or low-cost genetic testing to people with hemophilia and their families to identify ways to personalize patient care, generate new genetic information, and help create a databa

Alnylam Pharmaceuticals this week provided new guidance on its key drug-development programs, noting that its flagship transthyretin-mediated amyloidosis therapy ALN-TTR02 remains poised to enter phase III testing by the end of next year, and that the development of a subcutaneou

Alnylam Pharmaceuticals this week released new preclinical data on its GalNAc conjugate delivery technology, showing that the approach could permit subcutaneous delivery of siRNAs with a wide therapeutic index.

Alnylam Pharmaceuticals this week announced new preclinical data showing that its investigational RNAi-based treatment for hemophilia, ALN-AT3, could achieve target gene knockdown after subcutaneous administration to both mice and non-human primates.

Alnylam Pharmaceuticals announced this week that it has added ALN-APC, an siRNA-based treatment for hemophilia, to its formal drug-development pipeline.

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The Lancet has made changes to its peer-review process in response to its recent retraction of a COVID-19-related paper, Science reports.

The New York Times reports that a series of emails show how Department of Health and Human Services officials sought to silence the Centers for Disease Control and Prevention.

A new initiative aims to move Australia's genome sequencing labs onto one system, the Sydney Morning Herald reports.

In PLOS this week: recessive mutation tied to early-onset dilated cardiomyopathy, epigenetic analysis of lung adenocarcinoma, and more.