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hemophilia

Originally published Oct. 8

Alnylam Pharmaceuticals this week announced that it has received an orphan drug designation from the US Food and Drug Administration for its preclinical siRNA-based drug ALN-AT3 as a treatment for hemophilia A.

Officials from Alnylam Pharmaceuticals last week provided updates on the company’s pipeline programs, highlighting efforts involving its GalNAc conjugation technology, which enables subcutaneous siRNA delivery, and a fledgling effort in complement-mediated diseases.

Alnylam Pharmaceuticals this week announced that it has received an orphan drug designation from the US Food and Drug Administration for its subcutaneous hemophilia B treatment ALN-AT3.

Alnylam Pharmaceuticals this week released preclinical data showing that ALN-AT3, its investigational treatment for hemophilia and other bleeding disorders, can normalize thrombin generation and improve hemostasis in hemophilia mice and fully correct thrombin generation in a non-

Alnylam Pharmaceuticals this week announced that it plans to report initial data from an ongoing phase II trial of its lead drug candidate, the TTR-mediated amyloidosis treatment ALN-TTR02, this summer at an upcoming scientific meeting, and that it will launch an expansion study

Alnylam Pharmaceuticals this week provided an update to its research and development activities, adding a new preclinical program to its pipeline with ALN-AS1 for acute intermittent porphyria while dropping its refractory anemia drug ALN-HPN in order to focus on “higher priority”

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

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The Washington Post reports that a Russian Academy of Sciences commission has led to the retraction of hundreds of scientific papers.

The Los Angeles Times' Daily Pilot reports the chief executive of Vantari Genetics has pleaded guilty in a kickback scheme.

News 4 Jax reports that a Florida bill to prevent life and long-term care insurers from using genetic information in their coverage decisions has easily passed one committee.

In Science this week: potentially pathogenic mutations found in hematopoietic stem cells from young healthy donors, and more.