Roche's PiaSky has been recommended for use by the NHS in England and Wales for the rare blood disorder paroxysmal nocturnal haemoglobinuria (PNH), becoming the first option for patients th
Novartis' targeted Factor B inhibitor Fabhalta – the first oral monotherapy for rare disease paroxysmal nocturnal haemoglobinuria (PNH) – has been cleared by NICE for use by the NHS in Engl
Roche's PiaSky has been approved by the European Commission, becoming the first therapy for rare blood disorder paroxysmal nocturnal haemoglobinuria (PNH) that can be given by monthly subcu
AstraZeneca’s Voydeya has become the first oral Factor D inhibitor to be approved by the FDA as a treatment for the rare disease paroxysmal nocturnal haemoglobinuria (PNH)
The March meeting of the EMA’s human medicines committee (CHMP) saw an approval recommendation for Novartis’ Fabhalta, on track to become the first oral monotherapy for ra
In a new white paper from the World Without Disease initiative, a 2024 update is provided of the current endeavours and developments that have occurred since inaugural discussions last year