The FDA has cleared the IND for Ensoma's EN-374, targeting X-linked chronic granulomatous disease (X-CGD), a rare genetic ...

Abeona’s deal value is in line with recent voucher sale transactions amid uncertain times in rare disease drug development.

US President Donald Trump has signed an executive order telling drugmakers to cut prices to 'most favoured nation' pricing.

Mobile healthcare, web-based tech, and wearables are all contributing to a clinical trials landscape that puts the patient ...

GlobalData analysis predicts the global GLP-1RA market will reach $168bn by 2033, with Novo Nordisk and Eli Lilly to dominate ...

Orphan drug developers face significant difficulties when navigating market access frameworks in Europe. Could greater use of ...

The European Commission (EC) has granted orphan drug designation to Biodexa Pharmaceuticals’ oral tablet formulation of ...

The Trump administration has introduced legislation outlining plans for deep cuts to Medicaid, possibly impacting millions of ...

The UK MHRA has approved AstraZeneca's Trixeo Aerosphere inhaler to treat adults with chronic obstructive pulmonary disease ...

Eli Lilly and Purdue University, US, have announced the widening of their existing partnership with a planned investment of ...

The Danish multinational said that Sogroya improved the yearly growth rate in pre-pubertal children born small for ...

Minghui Pharmaceutical has announced an exclusive licensing and partnership agreement with Qilu Pharmaceutical for MHB088C in ...