FDA approved a once-daily dosing regimen for Orfadin nitisinone oral suspension and oral capsules from Swedish Orphan Biovitrum AB (SSE:SOBI) to treat hereditary tyrosinemia type I in patients ages ≥5 who have undetectable serum and...

New Therapeutic Targets and Biomarkers: July 2017 Select top therapeutic targets and biomarkers covered by BioCentury or added to the BCIQ database during July 2017. Therapeutic targets are defined as any protein, gene or other...

INDICATION: Acute myelogenous leukemia (AML) Patient sample, cell culture and mouse studies suggest inhibiting SYNCRIP could help treat AML. In bone marrow samples from patients, SYNCRIP levels were higher than in hematopoietic stem and progenitor...

A selection strategy commonly used to clone cells in vitro has been co-opted by a group at Oregon Health & Science University to create a gene therapy platform that gives a proliferation advantage to cells...

FDA approved an NDA from Swedish Orphan for an oral suspension formulation of Orfadin nitisinone to treat hereditary tyrosinemia type I, a rare genetic disorder that can cause liver failure, kidney dysfunction and neurological problems....

FDA accepted for review an NDA from Swedish Orphan for an oral suspension formulation of Orfadin nitisinone to treat hereditary tyrosinaemia type I, a rare genetic disorder that can cause liver failure, kidney dysfunction and...

The European Commission approved an oral suspension formulation of Orfadin nitisinone from Swedish Orphan to treat hereditary tyrosinaemia type I, a rare genetic disorder that can cause liver failure, kidney dysfunction and neurological problems. The...

When LSP-Life Sciences Partners launches a new healthcare venture fund next year it will stick to a strategy that has yielded 12 exits in the past 18 months - early stage, European drug development companies. The...