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Abstract
Introduction: Currently, there is no cure available for the hereditary neurodegenerative disease proximal spinal muscular atrophy (SMA), which is the number one genetic killer in early childhood. However, growing knowledge of SMA pathophysiology has opened new avenues for potential therapeutic interventions.
Areas covered: This review summarizes a variety of investigational therapeutic approaches for SMA. Focusing on the current state-of-the-art applications, the authors discuss the outcome of the first clinical interventions and compare the first results from the newest strategies. The achievements of the investigational drugs highlighted in this article were deduced from original articles, pharmaceutical company press releases and clinical trial results.
Expert opinion: Nearly two decades after the discovery of the disease causing gene survival motor neuron 1, many therapeutic options for SMA have been developed, some of which made it to clinical trials but could not prove their promising experimental results. Recently, big research efforts from academia, government and the pharmaceutical industry have led to the development of highly promising compounds that are currently in clinical trials, and which could lead to feasible treatment options in the future.
Acknowledgment
A Kaczmarek and S Schneider contributed equally to this work.
Declaration of interest
B Wirth is supported by the EU FP7/2007-2013 program Grant No 2012-305121 “NeurOmics”, the Deutsche Forschungsgemeinsschaft (Wi-945/14-2; Wi-945/16-1 and RTG 1960) and the Center for Molecular Medicine Cologne (C11). M Riessland is supported by SMA Europe. S Schneider and A Kaczmarek have been supported by the IGSDHD Graduate School for Biological Sciences.
Notes
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