News Myasthenia Gravis approval will boost Soliris revenues The FDA has approved Alexion's Soliris for use in patients with treatment resistant generalised Myasthenia gravis (gMG), making it the first new therapy for the condition in 60 years.
Views & Analysis Addressing market access obstacles for rare bleeding disorde... Scrutiniseing the market access situation for people with rare bleeding disorders.
News GSK’s ‘bubble baby’ gene therapy passes NICE’s new stricter ... GlaxoSmithKline’s gene therapy Strimvelis for the treatment of the ultra-rare ‘bubble baby syndrome’ has been approved by NICE.
News uniQure presses 'refresh' on haemophilia gene therapy, gets ... Biotech has been in talks with US and EU regulators
Views & Analysis Rare disease market research needs a multifaceted approach Challenges in rare disease market research and the role of online research.
News FDA experts to vote on Spark's groundbreaking gene therapy Luxturna could be single life-changing injection
Digital Post-ASCO 25: Pamela Tenaerts on CGTs and digital-first long... In a post-ASCO 2025 conversation, pharmaphorum web editor Nicole Raleigh spoke with Dr Pamela Tenaerts, chief medical officer at Medable.
Market Access The value of a partner, globally and locally A new podcast looks at recent trends related to drug approvals, as well as challenges and opportunities both small and large pharma companies face