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.

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

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

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

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

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 new site will support Roche and its unit Genentech's future portfolio of next-generation obesity medicines.

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

The executive order aims to cut US prescription drug costs by linking them to the lowest prices paid internationally.

Both the FDA and EMA have suspended Ixchiq’s use in older adults after post-marketing serious adverse events that include two ...