An exploratory randomised double-blind and placebo-controlled phase 2 study of a combination of baclofen, naltrexone and sorbitol (PXT3003) in patients with Charcot-Marie-Tooth disease type 1A.

Orphanet J Rare Dis · 2014 · PMC4311411 · PMID 25519680

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showed a trendno p-value reported

Among the functional measures (including 6MWT, 9HPT, Ankle Dorsiflexion and Hand Grip), 6MWT and Hand Grip showed a trend toward improvement in the HD group compared to PLI despite evidence of some training effect assessed from the Placebo group and PLI.

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