Sanofi has claimed approval in the EU for Xenpozyme as a treatment for acid sphingomyelinase deficiency (ASMD) – which becomes the first and only treatment option for patients with the rare
Shares in Sarepta came under pressure after the biotech said it had temporarily halted a clinical trial of its new-generation Duchenne muscular dystrophy candidate, after a patient had a se
Last year, AstraZeneca reported that a new drug to treat Wilson disease developed by its Alexion rare diseases unit had hit the mark in a phase 3 trial, setting it on course for regulatory
Armed with new phase 3 data, AstraZeneca is preparing to file for regulatory approval of eplontersen, an antisense drug for one of the complications of the disease transthyretin amyloidosis
bluebird bio started last week on tenterhooks, as FDA advisors cast their eyes over data for two gene therapy candidates that are key to its future as a commercially viable concern.
A pharma company’s core mission is to improve patient outcomes. This hinges on effectively influencing HCP clinical behaviour and driving disease education.
