Exciting news

Posted on: 08/06/2016

Following 20 successful transplants in Milan, Gene Therapy for MLD is now being considered for European license. This means that, if successful, hospitals everywhere will be able to transplant children as soon as they are diagnosed - providing that their diagnosis comes before the disease is starting to progress. Italy have already been granted their license and we hope the UK license will follow soon! This is wonderful news and an incredible step towards combating this horrendous disease. That said, the sad reality is that most children are diagnosed too late to receive treatment. With that in mind, a research team led by Dr Michael Gelb at the University of Washington has been diligently working for the past few years on devising a test for MLD to be administered to all newborn babies. A few months ago Dr Gelb announced that he had devised a successful kit and last week he commenced a live pilot in the US to prove his theory. This groundbreaking work is the first step towards getting MLD accepted onto the newborn heel prick blood test in the UK. We at ArchAngel are now dedicating our efforts towards making this happen and I am very excited to be driving this campaign forward. Tomorrow I have a meeting with the PANS (Patient Advocates for Newborn Screening) group in Manchester and I look forward to updating you with more news about this life saving campaign.