The National Hospital and Institute of Neurology is a world leading centre of excellence for clinical care and research into neurological diseases.
We are a multidisciplinary centre for patients with neuromuscular diseases, combining patient care, training and research.
We provide comprehensive NHS clinical services for over 5,000 patients per year which include a range of specialist neuromuscular clinics and nationally commissioned services.
We provide national and international clinical and scientific training programmes for doctors, nurses, physiotherapists and allied healthcare professionals.
We undertake major programmes of externally funded basic science and translational research to find therapies and improve the lives of patients.
We prioritise national and international partnership' working with NHS and academic colleagues, industry, patient organisations and patients.
Current trials- SIDEROS (DMD)
Past trials – OLEOS for SMA, Arimoclamol for IBM, Bumetanide for Hypopp, Sodium Valproate for McArdle disease, dichlorphenamide for periodic paralysis
The Centre for Neuromuscular disease has been awarded MRC funding for translational research in Neuromuscular disease since 2008 and has recently successfully obtained further funding.
|Study Name||Trial Status at Queens Square||Overall Trial Status|
|Santhera (SIDEROS)||Fully recruited||Fully recruited|
I have been a consultant in Neuromuscular disease since 1995, and have worked at Queen Square since 2008. I am the clinical lead for the neuromuscular complex care centre and have over 100 adult Duchenne patients under my care.
I was a PI for the phase 2b randomised controlled trial of Ataluren in Duchenne muscular dystrophy (DMD) and am currently a PI for the Sideros study which is evaluating the effect of idebenone in corticosteroid treated DMD patients. I am the lead for the adult North Star Network which is establishing a national database for adults with DMD to understand the natural history of this patient population. I am joint co-ordinating editor for the Cochrane Neuromuscular disease Group.