News Sarepta dives deeper into gene therapy Sarepta Therapeutics has handed $30 million to Lacerta in exchange for a group of gene therapies, including a Pompe disease candidate.
News Summit shares plunge nearly 80% after DMD drug fails Summit Therapeutics has axed development of its Duchenne muscular dystrophy drug ezutromid after a spectacular failure in phase 2, causing the UK biotech's shares to plunge.
News Pharma collaborates to improve chances of NICE backing Duche... Project will initially focus on DMD but could benefit other rare diseases
News Contrasting fortunes for Duchenne rivals, as PTC fails and S... Two rivals in the emerging Duchenne muscular dystrophy (DMD) therapy area have experienced contrasting fortunes this week.
News PTC Therapeutics fails to convince FDA panel about DMD drug FDA advisers have voted against approval for PTC Therapeutics’ Duchenne drug Translarna because of doubts about its efficacy.
News FDA reviewers slam PTC's Duchenne drug ahead of meeting FDA staff not persuaded by patchy clinical trial evidence for Translarna.
News Agios mulls early FDA filing for sickle cell therapy Agios is planning to file its sickle cell disease candidate mitapivat with the FDA under the accelerated pathway within the next few months.
Sales & Marketing Sponsored Using AI-driven synthetic personas to take your insights fur... As AI continues to reshape the healthcare landscape, pharma teams are beginning to leverage synthetic personas.
