TPP1 and proximal spinal muscular atrophy: While in recent years, there have been few successes in the translation of research to therapies, notably for monogenetic conditions such as gene-targeted treatments for Spinal Muscular Atrophy (SMA) and the CLN2 form of Batten disease [1,2,3,4,5,6,7] and oral supplementation therapy for hereditary sensory and autonomic neuropathy disease (HSAN1) [8], it remains even more difficult to design targeted therapies for the majority of idiopathic non-heritable neurodegenerative conditions.