Alexion is a somewhat unique pharma company in that it focuses almost entirely on rare diseases – and although ... including AstraZeneca, Novartis and Allergan. About the author Dr Paul Tunnah ...

AstraZeneca’s rare disease firm Alexion is set to expand its genomic medicine portfolio with the acquisition of gene editing specialist LogicBio Therapeutics, in a deal worth approximately $68 ...

Company to assess alternate payloads; no changes planned to novel capsid component - - Resulting shift of timeline and costs extends cash ...

LEXINGTON, Mass., Feb. 06, 2025 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today announced it will ...

The Rare Disease Network, which includes Calvin University, Corewell Health Helen DeVos Children’s Hospital, MSU College of Human Medicine, Pine Rest Christian Mental Health Services and Van Andel ...

The FDA has made some modest efforts, but it needs an internal organization that understands and would be an aggressive advocate for the development of rare-disease drugs. When I joined the FDA to be ...

Some of our programs are wholly owned, and some are advancing with partners including Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; and Neurocrine Biosciences, Inc. For more information ...

Sopra Steria is one of the only five providers to achieve a Best-in-class overall rating in 2024 as supplier of ServiceNow-related servicesPAC highlighted Sopra Steria's ability to extend ServiceNow ...

today announced that it has entered into an agreement with Alexion, AstraZeneca Rare Disease for an exclusive worldwide license to ALXN-1840 (bis-choline tetrathiomolybdate), a drug candidate for ...