Dexpramipexole shows promise for ALS in phase II trial

Kingwell, Katie

doi:10.1038/nrneurol.2011.204

Research Highlight
Published: 26 December 2011

Neurodegenerative disease

Dexpramipexole shows promise for ALS in phase II trial

Katie Kingwell

Nature Reviews Neurology volume 8, page 4 (2012)Cite this article

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The putative mitochondrial modulator dexpramipexole seems to slow functional decline and might reduce mortality in patients with amyotrophic lateral sclerosis (ALS), according to a phase II study.

ALS is a fatal neurodegenerative disease, for which riluzole is the sole approved treatment and only extends patient survival by a matter of months. A previous open-label study had suggested a trend towards attenuated functional decline in patients treated with dexpramipexole.

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ORIGINAL RESEARCH PAPER

Cudkowicz, M. et al. The effects of dexpramipexole (KNS-760704) in individuals with amyotrophic lateral sclerosis. Nat. Med. doi:10.1038/nm.2579
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