Research and Innovation

Novel therapy for Battens Disease available on the NHS

Patients at Great Ormond Street Hospital (GOSH) and across England are set to receive treatment for the rare nervous system condition Battens Disease, following a deal between the NHS and manufacturer Biomarin. The drug, which is called cerliponase alfa or Brineura, has been approved by the NHS following successful clinical trials at GOSH in collaboration with other centres from across the world. 

How 3d modelling and virtual reality can benefit patients and staff ahead of surgery

​Joe, aged 15, who features on Paul O’Grady’s Little Heroes, is being treated at GOSH for Marfan Syndrome, a genetic disorder that affects the body’s connective tissue, which plays an important role in helping the body grow and develop properly. Joe has an associated heart condition and required surgery to replace his aorta, the body’s main artery. 

The medical and research team involved in his care share how they have used pioneering 3D Heart Modelling and Virtual Reality to aid understanding of his heart condition ahead of surgery. 

European clinical trial for Kawasaki disease to be led by GOSH and ICH researchers

Researchers at the UCL Great Ormond Street Institute of Child Health (ICH) and Great Ormond Street Hospital (GOSH) are to lead a new clinical trial for Kawasaki syndrome (KD-CAAP), a rare immune system condition that causes swelling of the blood vessels of the heart and can lead to severe fever and risk of heart problems such as aneurysms.