Strimvelis

News

GlaxoSmithKline’s gene therapy Strimvelis for the treatment of the ultra-rare ‘bubble baby syndrome’ has been approved by NICE.

News

Ultragenyx's therapy for the rare genetic disorder Angelman syndrome, apazunersen, has flunked a late-stage trial, crashing its share price.

Digital

A new webinar from Konovo will address some of the critical topics impacting today’s healthcare market research industry.