hemophilia

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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.

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.

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Sometimes genetic tests give inconclusive results and provide little reassurance to patients, the Associated Press reports.

Vox wonders whether gene-editing crops will be viewed similarly as genetically modified organisms of if people will give them a try.

In Science this week: research regulation and reporting requirement reform, and more.

With H3Africa, Charles Rotimi has been working to bolster the representation of African participants and African researchers in genomics, Newsweek reports.