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Multiple system atrophy (MSA), a rapidly progressive neurodegenerative disorder with parkinsonism, cerebellar ataxia, and autonomic failure, has few effective therapies, so disease-modifying therapies remain a critical need. So far, randomized controlled trials (RCTs) testing a variety of drug candidates largely failed.1 The exception was a small-scale interventional RCT of sequential intra-arterial and IV administration of mesenchymal stem cells (MSC) in patients with cerebellar-type MSA2 that found a small reduction in the increase of severity rating scores (Unified MSA Rating Scale [UMSARS]) over 1 year, as compared to placebo.
Footnotes
See page 24
Disclosure: F. Krismer reports a research grant from the MSA Coalition. K. Seppi reports personal fees from Teva, UCB, Lundbeck, AOP Orphan Pharmaceuticals AG, Roche, Grünenthal, and AbbVie; honoraria from the International Parkinson and Movement Disorders Society; and research grants from FWF Austrian Science Fund, Michael J. Fox Foundation, and International Parkinson and Movement Disorder Society, outside the submitted work. W. Poewe has received consultancy fees from Astra Zeneca, Lundbeck, and Biogen in relation to MSA drug development; has received personal fees from AbbVie, Allergan, Astra Zeneca, BIAL, Biogen, Boehringer‐Ingelheim, Boston Scientific, GlaxoSmithKline, Ipsen, Lundbeck, Medtronic, MSD, Merck‐Serono, Merz, Novartis, Orion Pharma, Teva, UCB, and Zambon, outside the submitted work; and has received royalties from Wiley Blackwell, Oxford University Press, and Cambridge University Press. Go to Neurology.org/N for full disclosures.
- © 2019 American Academy of Neurology
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