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.
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sickle cell disease
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".
6 December 2016 | By Niamh Louise Marriott, Digital Editor
Results from the Phase II study show that SEG101 an anti-P-selectin antibody, reduced the median annual rate of sickle cell-related pain crises by 45.3%...
27 June 2016 | By Victoria White, Digital Content Producer
The Angola Sickle Cell Initiative and Bristol-Myers Squibb are teaming up to provide children with sickle cell disease in Angola with Droxia...
26 May 2016 | By Victoria White, Digital Content Producer
A Managed Access Program (MAP) for the treatment of sickle cell disease has been announced for Emmaus’ pharmaceutical grade L-glutamine treatment (PGLG)...