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- Title
A meta-analysis of randomized double-blind clinical trials in CMT1A to assess the change from baseline in CMTNS and ONLS scales after one year of treatment.
- Authors
Mandel, Jonas; Bertrand, Viviane; Lehert, Philippe; Attarian, Shahram; Magy, Laurent; Micallef, Joëlle; Chumakov, Ilya; Scart-Grès, Catherine; Guedj, Mickael; Cohen, Daniel
- Abstract
CMT1A is the most common inherited peripheral neuropathy. There is currently no approved treatment. We performed a meta-analysis including four randomized, double-blind, Placebo-controlled clinical trials to assess the disease progression after one year under Placebo, Ascorbic Acid (AA) or PXT3003, a combination of three repurposed drugs. We observed a weak deterioration in patients under Placebo, well below the reported natural disease progression. Patients treated with AA were stable after one year but not significantly different from Placebo. Patients undergoing PXT3003 treatment showed an improvement in CMTNS and ONLS, statistically significant versus Placebo and potentially precursory of a meaningful change in the disease course.
- Subjects
CHARCOT-Marie-Tooth disease; PERIPHERAL neuropathy; META-analysis; CLINICAL trials; PLACEBOS
- Publication
Orphanet Journal of Rare Diseases, 2015, Vol 10, Issue 1, p1
- ISSN
1750-1172
- Publication type
Article
- DOI
10.1186/s13023-015-0293-y