For decades, treatment options for rare diseases have been limited, often focusing on symptom management rather than addressing the root cause of them
Sanofi is preparing a phase 3 programme for asthma candidate amlitelimab, despite mixed data in a phase 2 trial.
Shares in Ironwood fell by a third after it revealed the FDA is insisting on a new phase 3 trial of its short bowel syndrome therapy apraglutide.
Bristol-Myers Squibb's Camzyos has fallen short in a trial in non-obstructive hypertrophic cardiomyopathy, denting its growth prospects.
Scancell is using a new service launched by the NHS to pair patients with suitable clinical trials in a phase 2 trial of its melanoma cancer vaccine.
Editor's Picks
