NICE recommends once-weekly injection for severe blood clotting disorder Efanesoctocog alfa, a once-weekly treatment for patients with severe haemophilia A, removes the need for frequent dosing, owing to its effective bleeding control.…
NICE approves gene-editing sickle cell therapy for NHS in England Exagamglogene autotemcel, also known as exa-cel, is now recommended for NHS use, reversing previous draft guidance.…
Manufacturer withdraws treatment for sickle cell disease following concern over deaths Pfizer's withdrawal is based on data that suggest “an imbalance in vast-occlusive crises and fatal events which require further assessment”.…
First gene-editing therapy recommended by NICE to treat people with beta thalassaemia NHS England said that around 460 patients aged 12 years and over could benefit from the treatment.…
Haemophilia treatment first to be recommended by NICE as part of new drug pricing agreement Etranacogene dezaparovovec (Hemgenix; CSL Behring) is also the first medicinal product to enter the Innovative Medicines Fund.…
NICE recommends voxelotor for sickle cell treatment, reversing previous decision The National Institute for Health and Care Excellence said it was able to recommend the sickle cell treatment following a reduction in its price.…
Iron deficiency anaemia: symptoms, diagnosis and management Globally, iron deficiency anaemia is the most common type of anaemia and has numerous causes. Pharmacists should be aware that patients can present with a range of symptoms.…
Gene-editing sickle cell drug not approved for NHS use in draft NICE guidance The National Institute for Health and Care Excellence said it required “additional data” beyond the current proposal.…
NICE requests more evidence on sickle cell disease treatment voxelotor The National Institute for Health and Care Excellence initially rejected voxelotor for NHS use on the grounds that clinical trial evidence did not support cost effectiveness.…
Our drive to increase diversity on the stem cell register How a partnership between the Muslim Doctors Cymru group and charity Anthony Nolan is promoting diversity in stem cell donations.…
