
A new Clinical Trial has been announced by Shire (now part of Takeda Pharmaceutical Company Ltd) for Intrathecal Enzyme Replacement Therapy for children with Late-Infantile MLD. The centre in the UK will, hopefully, be Bristol Royal Hospital for Children. Commencement will not be earlier than late 2019, and they will be recruiting children with a confirmed diagnosis of Late-Infantile MLD, who are either pre-symptomatic or symptomatic, but who have had no previous treatment such as Stem Cell Transplantation or Gene Therapy. Further details will be available later in the year.