About University of Sheffield
On 28 February we will be launching a fundraising appeal for our research into rare genetic diseases. We have a team of leading specialists who are experienced at developing gene therapy treatments for genetic diseases including Spinal Muscular Atrophy. We need to raise money to help fund more of these research projects, including for COL4A1, SYNGAP1, AP4 (SPG47) and many others. Please get in touch if we can work together in any way? We’ll be doing lots on our social media channels to raise awareness, share patient stories and information from our academics.