Alnylam Pharmaceuticals said this week that the US Food and Drug Administration has given orphan drug status to the company's investigational transthyretin-mediated amyloidosis ALN-TTR02 as a treatment for familial amyloidotic polyneuropathy, a common manifestation of the disease.

The designation provides a drugmaker with, among other things, extended market exclusivity for its product, tax credits, and marketing incentives.

Get the full story with
GenomeWeb Premium

Only $95 for the
first 90 days*

A trial upgrade to GenomeWeb Premium gives you full site access, interest-based email alerts, access to archives, and more. Never miss another important industry story.

Try GenomeWeb Premium now.

Already a GenomeWeb Premium member? Login Now.
Or, See if your institution qualifies for premium access.

*Before your trial expires, we’ll put together a custom quote with your long-term premium options.

Not ready for premium?

Browse our free articles
You can still register for access to our free content.

In Science this week: factors influencing retrotransposon integration sites, and more.

A bioethicist argues for the responsible use of germline gene editing.

Some breweries are using DNA-based testing to determine whether unwanted bacteria are affecting their beers, The Verge reports.

Standardized N-of-1 trials will be needed to test out personalized medicines, writes Nicholas Schork from the J. Craig Venter Institute at Nature.