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Patients with rare blood disorders to receive new treatment option, says NICE
In final guidance published today (20 May 2021), NICE has recommended ravulizumab (Ultomiris, Alexion Pharmaceuticals) as a treatment for paroxysmal nocturnal haemoglobinuria (PNH). Draft guidance published 19 May 2021 also recommends the treatment for atypical haemolytic uraemic syndrome (aHUS).
New life-changing treatment option recommended for type of rare epilepsy
This marks a significant step forward for around 1,400 people living with this rare and severe form of epilepsy, which typically begins in early childhood.