BioMarin would like to announce that Great Ormond Street Hospital (GOSH) in London will be joining the European clinical study centres participating in the BioMarin BMN190 trial in the immediate future. At the time when GOSH initiates the study, the…
Batten Disease Family Association welcomes Neurological Alliance’s five calls to action for prioritising neurological services The Neurological Alliance has today launched a new report, Going the distance: National calls to action to drive neurological service improvement in England, taking a…
Improving Rare Disease Education and Training for UK Nurses: hugely important initiative to ensure earlier diagnosis and better focussed care. The British government recently launched a first-of-its-kind national initiative, “UK Strategy for Rare Diseases,” to promote earlier diagnosis and treatment…
The Evelina London Children’s Hospital, Guy’s and St Thomas’ NHS Foundation Trust, London has begun recruiting subjects with late infantile CLN2 disease, a form of Batten disease, for a clinical trial. The trial will examine whether BMN190, an experimental drug,…
The BDFA are delighted to welcome Professor Paul Gissen to our team of Medical and Scientific Advisors. Professor Gissen is a Consultant in Inherited Metabolic Diseases (IMDs) at Great Ormond Street Hospital for Children, London, UK and a Wellcome Trust Senior Research…