Sebastian Stachowiak, Head of Europe & GCC at Global Blood Therapeutics (GBT), explains how, while the company establishes its presence in Europe, it continues its commitment to advance the care of people living with sickle cell disease by collaborating with the sickle cell community to develop breakthrough therapies, raise awareness…
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sickle cell disease
The gene therapy corrected the shape of some sickle cell patients' red blood cells and eliminated episodes of severe pain.
Crizanlizumab recommended as a treatment option for preventing recurrent sickle cell crises for people aged 16 and over in the UK.
To promote the safe and ethical use of genome editing for the treatment of disease and genetic disorders WHO has published the first global recommendations.
Data shows treating sickle cell disease patients aged four to 11 years with Oxbryta® (voxelotor) results in increases in haemoglobin associated with improved clinical status.
Adam Pearson, Senior Oncology Analyst at GlobalData, comments on the development of CRISPR-based therapies and outlines potential opportunities and challenges in the space.
The Gates Foundation will fund Novartis’ discovery and development of a gene therapy for sickle cell disease that is accessible for low- and middle-income countries.
The DECODR app was developed to accelerate the development of CRISPR gene therapies, producing similar data as deep sequencing processes in a shorter timeframe and at less cost.
The EMA has accepted Global Blood Therapeutics' marketing authorisation application for Oxbryta (voxelotor) to treat haemolytic anaemia in sickle cell disease.
Dapivirine Vaginal Ring (dapivirine) and Blenrep (belantamab mafodotin) have been recommended for approval by the EMA's CHMP, along with nine other medicines.
New research from PhRMA found that in the US, there are almost 600 paediatric medicines in development in over 2,000 clinical trials.
Adakveo has been approved by the FDA to reduce the frequency of vaso-occlusive crisis, a painful complication of sickle cell disease.
A new liquid formulation of hydroxycarbamide to treat sickle cell disease will “enable doctors to personalise doses in children".