hemophilia

Spark Therapeutics reports new data on its investigational hemophilia A treatment that says it reduces bleeds, according to Stat News.

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Gene therapies could qualify for a faster US Food and Drug Administration approval process, according to Stat News.

The free genetic testing program has amassed genetic data and samples from more than 5,000 patients that researchers can now apply to access.

The firm also said it aims to soon begin human studies for its hepatic porphyria treatment ALN-AS1 and to select a development candidate for its program in primary hyperoxaluria type 1.

The therapy is the company's first to feature an optimized version of its GalNAc conjugate delivery technology.

NEW YORK (GenomeWeb) – As Phase I testing of its hemophilia therapy ALN-AT3 continues, Alnylam Pharmaceuticals is setting its sights broadly for the program, taking aim at both patients receiving standard medical care as well as those who have become refractory to such treatment, company official

NEW YORK (GenomeWeb) – Although Alnylam Pharmaceuticals has focused on rare, genetically defined diseases in recent years, the company sees opportunities to someday move back into the broader indications that once populated its research and development initiatives, a top company official said las

NEW YORK (GenomeWeb) – Alnylam Pharmaceuticals this week announced that its Phase I hemophilia drug ALN-AT3 has received orphan drug status from European regulators.

Alnylam Pharmaceuticals this week announced that it has begun a phase I trial of ALN-AT3, an RNAi-based treatment for hemophilia and other bleeding disorders.

Alnylam Pharmaceuticals this week announced that it has filed a clinical trial application with UK regulators to begin human testing of an siRNA-based treatment for hemophilia A and B.

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Senator Elizabeth Warren (D-Mass.) has released the results of a genetic ancestry analysis, the Boston Globe reports.

Retraction Watch's Ivan Oransky and Adam Marcus report that Harvard Medical School and Brigham and Women's Hospital have recommended that more than 30 papers from a former researcher be retracted.

Thomas Steitz, who won the 2009 chemistry Nobel Prize for his ribosome work, has died, the Washington Post reports.

In PLOS this week: mechanisms for genes implicated in coronary artery disease, rumen microbes and host genetics influence cow methane production, and more.