Two Peas in a Pod
Last weekend we attended first ever conference for MLD in the UK. I was privileged to be invited to the scientific meeting on day 1, attended by some exceptional medical professionals, including metabolic expert Prof. Tim Cox; Brains 4 Brain founder Dr Brian Begley; Head of Laboratories at GOSH Simon Heales; and paediatric neurologist Dr Ming Lim. It was fantastic to hear them share their experiences and knowledge of the disease and also enlist their support for our newborn screening campaign. Day 2 was family day and although we didn’t quite get all of the affected UK families in the same room, it was excellent to meet several of them. It was desperately sad to see the children who are too affected by the disease to qualify for Gene Therapy, but the courage and positivity shown by their parents was truly inspirational. Ava was happy to be reunited with her GT buddy Talleulah, whom she met in Milan in between their respective treatments. We were all amazed to see how similar the girls were in every mannerism and it was lovely to see their closeness, as they clearly understand their unique bond. Angelina enjoyed meeting the other families too and it was good for her to feel that she is not the only one with a sibling in such an unusual situation. Two of the ArchAngel Trustees, Laura and Rachael, also attended and gave an excellent presentation about our work and the grants which we have available to help families. They also distributed very generous and thoughtful gifts to the affected children and families and I felt extremely proud of the palpable compassion and kindness which they brought to the event. Well done to the MLD Support Association UK for organising such a worthwhile conference. I hope it is the first of many.