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Following news today that medical regulators have approved a gene therapy with the potential to cure the blood disorders sickle cell disease and beta thalassemia, Yasmin Sheikh, Head of Policy and Public Affairs at Anthony Nolan, said:
“We’re delighted this world-first therapy has been granted an MHRA license, which brings us a step closer to it being available to people with severe sickle cell disease in the UK.
"Patients won’t be able to access Casgevy on the NHS until it’s approved by NICE. We hope that NICE will listen to patients with severe sickle cell disease who desperately need better treatment options and make this innovative therapy available as quickly as possible.”