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Efficacy of N-163 strain of Aureobasidium pullulans-produced beta-glucan in improving muscle strength and function in patients with Duchenne muscular dystrophy; Results of a 6-month non-randomised open-label linear clinical trial

Raghavan, K.; Sivakumar, T.; Bharatidasan, S. S.; Srinivasan, S.; Dedeepiya, V. D.; Ikewaki, N.; Senthilkumar, R.; Preethy, S.; Abraham, S. J.

2023-05-02 neurology
10.1101/2023.04.29.23289260 medRxiv
Show abstract

Duchenne muscular dystrophy (DMD) is a debilitating genetic disease that causes gradual muscle weakening and early mortality. After a 45-day clinical pilot study and a pre-clinical study in mdx mice demonstrating safety and efficacy, and anti-fibrotic and anti-inflammatory effects, respectively, of an Aureobasidium pullulans N-163 strain-produced B-1,3-1,6-glucan. We conducted this linear six-month clinical study to assess its efficacy. Twelve ambulatory and 12 non-ambulatory individuals with DMD were included; all received the N-163 strain of A.pullulans produed beta-glucan dietary supplement orally in addition to the standard treatment regimen, which included steroids. The Medical Research Council muscle score improved in 11 patients in the ambulatory group and in 8 patients in the non-ambulatory group. The six-minute walk test distance improved in nine patients, with a 29.5-meter average improvement. The North Star Ambulatory Assessment improved by 1 unit in three patients. This safe beta-glucan food supplement improved muscle function within 6 months. A comprehensive, multi-centric clinical research should be conducted for unravelling its potential as a disease-modifying drug adjuvant in DMD.

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