The newly established UCL Queen Square Motor Neuron Disease and the Leonard Wolfson Experimental Neurology Centres (the UK’s first dedicated Clinical Research Facility designed to fast-track translational research and therapeutic innovation in neurodegeneration) bring together world-leading research groups spanning the spectrum of basic discovery science, translational and experimental medicine clinical trials. This partnership has led to the consolidation of a strong portfolio of ground-breaking trials prioritizing immunotherapies, cell and gene therapies, and single gene antisense oligonucleotide (ASO) trials by intrathecal administration. Engaging with national and international partners and the UK’s leading centre for multidisciplinary training of basic and clinical scientists in MND, this partnership is ideally placed to support novel therapeutic developments in ALS. UCL Queen Square Institute of Neurology (IoN) has significant research and clinical expertise in Motor Neuron Disorders, including Amyotrophic lateral sclerosis (ALS) and Kennedy’s Disease (KD; also known as Spinal Bulbar Muscular Atrophy – SBMA).
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