Alnylam Pharmaceuticals
NEWS
The top three clusters from last year’s list - Greater Boston, San Francisco Bay Area and San Diego - all stood their ground and maintained their rankings from the previous year.
While the study hit its primary efficacy endpoints, investors are likely concerned about reports of “inconsistent results” regarding reducing Uox excretion in a subtype of patients with the rare disease.
Tadataka “Tachi” Yamada, a former GlaxoSmithKline and Takeda Pharmaceutical executive, early gene therapy backer, and philanthropist, passed away on Wednesday of natural causes at the age of 76.
The FDA has already given its go-ahead to use lumasiran to treat PH1 and lower the urinary oxalate levels in pediatric and adult patients under the brand name OXLUMO.
Interim results from the Phase I study presented at the Peripheral Nerve Society Annual Meeting demonstrated that the investigational therapy, NTLA-2001, greatly reduced the disease-causing protein after a single infusion.
The U.S. FDA has had a mix of announcements this week, from drug approvals to the acceptance of NDAs and INDs. Here’s a look.
BioSpace looks at five of the top investor companies that support life sciences companies across the industry.
The Rare Disease Company Coalition’s 10 founding members have brought 22 treatments to market and currently have more than 160 rare disease programs in the works.
The new year began with a fairly low level of clinical trial news. Here’s a look.
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