SCIDNET is a European Commission Horizon 2020 -funded project which launched in January 2016. It aims to take gene therapy from current clinical trials to a medicinal product that can be delivered to any child in the EU. It follows on from previous EU col
The importance of prognostic biomarkers in improving risk stratification in Wilms’ Tumour
16 Sep 2016, 11:03 a.m.
Research led by BRC funded Professor Kathy Pritchard-Jones has demonstrated the importance of prognostic biomarkers in improving risk stratification in Wilms’ Tumour (WT).
NICE recommend drug trialled at GOSH for the treatment of Duchenne Muscular Dystrophy
16 Sep 2016, 11:02 a.m.
NICE have published final guidance recommending ataluren for the treatment of Duchenne Muscular Dystrophy (DMD) caused by a nonsense mutation, in children aged 5 years and over who are able to walk.
Research identifies novel intracellular pathway important for collagen homeostasis
16 Sep 2016, 11 a.m.
A collaboration between researchers at UCL, led by BRC-funded Professor Paul Gissen, has identified a novel intracellular pathway important for generic collagen homeostasis. Furthermore, this pathway was found to be dependent on two proteins that are defe
Identification of defective gene causing a form of childhood Parkinsonism
16 Sep 2016, 10:58 a.m.
Researchers led by BRC-supported Dr Karin Tuschl in collaboration with Dr Esther Meyer have discovered that a form of childhood Parkinsonism is caused by a defective gene encoding a metal transporter called SLC39A14