Emerging Therapies

Two large, phase 3 clinical trials demonstrated that targeting B-cells can have a significant impact on disease progression in patients with relapsing multiple sclerosis.
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Although multiple sclerosis (MS) is dominated by a progressive phase of the disease, few therapies are available to modify clinically defined progression. There is a significant unmet need for treatments targeting the delayed neurodegenerative components of the disease, said Gavin Giovannoni, MBBCh, PhD, Chair of Neurology, Blizard Institute of Cell and Molecular Science, Queen Mary University of London, England, at the 2016 American Academy of Neurology annual meeting.

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A pivotal phase 3 clinical trial demonstrated that dichlorphenamide (Keveyis) significantly reduced the rate and severity of hypokalemic episodes in patients with periodic paralysis, a rare muscle disease affecting children and young adults. In addition, a parallel phase 3 clinical trial with dichlorphenamide showed a similar treatment effect in patients with hyperkalemic periodic paralysis but failed to demonstrate significance because of recruitment issues.

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