PDF(Pubmed)
Abstract：
UNASSIGNED: Muscular dystrophies other than Duchenne muscular dystrophy (DMD) are genetic diseases characterized by increasing muscle weakness, loss of ambulation, and ultimately cardiac and respiratory failure. There are currently no effective therapeutics available. Having demonstrated the efficacy of a N-163 strain of Aureobasidium Pullulans (Neu-REFIX) produced B-1, 3-1,6-Glucan in pre-clinical and clinical studies of Duchenne muscular dystrophy (DMD) earlier, we assessed the effectiveness of this novel Beta glucan in the other muscular dystrophies in the present study.
UNASSIGNED: In this 60-day study, six patients with muscular dystrophies other than DMD consumed one 8g gel of Neu-REFIX beta-glucan along with their usual standard of care treatment regimen, and their biomarkers of relevance to muscle function such as serum calcium (SC), creatine phosphokinase (CPK), and alkaline phosphatase (ALP) levels along with functional improvement criteria, which is, Medical research council (MRC) scale and North Star Ambulatory assessment (NSAA), assessed at baseline and following the intervention.
UNASSIGNED: After the intervention, the SC levels significantly decreased from a mean baseline value of 9.28 mg/dL to 8.31 mg/dL (p-value = 0.02). With a p-value of 0.29, the mean CPK value dropped from 2192.33 IU/L to 1567.5 IU/L. Following the intervention, the ALP levels dropped from 200.33 to 75.5 U/L (p-value = 0.15). MRC scale improved in three out of six patients. NSAA remained stable. There were no adverse effects.
UNASSIGNED: This study has proven the safety of Neu REFIX beta-glucan food supplement and its efficacy in improving both plasma biomarkers and functional parameters of muscle in a short duration of 2 months. Further validation by evaluation of muscle function for a longer duration is recommended to confirm the efficacy of Neu-REFIX food supplement as a potential adjuvant DMT in muscular dystrophies.
UNASSIGNED: In this 60-day study, six patients with muscular dystrophies other than DMD consumed one 8g gel of Neu-REFIX beta-glucan along with their usual standard of care treatment regimen, and their biomarkers of relevance to muscle function such as serum calcium (SC), creatine phosphokinase (CPK), and alkaline phosphatase (ALP) levels along with functional improvement criteria, which is, Medical research council (MRC) scale and North Star Ambulatory assessment (NSAA), assessed at baseline and following the intervention.
UNASSIGNED: After the intervention, the SC levels significantly decreased from a mean baseline value of 9.28 mg/dL to 8.31 mg/dL (p-value = 0.02). With a p-value of 0.29, the mean CPK value dropped from 2192.33 IU/L to 1567.5 IU/L. Following the intervention, the ALP levels dropped from 200.33 to 75.5 U/L (p-value = 0.15). MRC scale improved in three out of six patients. NSAA remained stable. There were no adverse effects.
UNASSIGNED: This study has proven the safety of Neu REFIX beta-glucan food supplement and its efficacy in improving both plasma biomarkers and functional parameters of muscle in a short duration of 2 months. Further validation by evaluation of muscle function for a longer duration is recommended to confirm the efficacy of Neu-REFIX food supplement as a potential adjuvant DMT in muscular dystrophies.
摘要：
■除Duchenne肌营养不良（DMD）以外的肌营养不良是以肌肉无力增加为特征的遗传性疾病，失去行走能力，最终导致心脏和呼吸衰竭.目前没有有效的治疗方法。在早期Duchenne型肌营养不良症（DMD）的临床前和临床研究中，已经证明了N-163株Pullulans（Neu-REFIX）产生的B-1，3-1,6-葡聚糖的功效，在本研究中,我们评估了这种新型β-葡聚糖在其他肌营养不良中的有效性.
■在这项为期60天的研究中，6例除DMD以外的肌营养不良患者服用了一种8克Neu-REFIXβ-葡聚糖凝胶，以及他们通常的标准治疗方案，以及它们与肌肉功能相关的生物标志物，如血清钙（SC），肌酸磷酸激酶（CPK），和碱性磷酸酶（ALP）水平以及功能改善标准，也就是说，医学研究委员会（MRC）量表和北极星门诊评估（NSAA），在基线和干预后进行评估。
■干预后，SC水平从平均基线值9.28mg/dL显著降低至8.31mg/dL(p值=0.02).p值为0.29时，CPK的平均值从2192.33IU/L降至1567.5IU/L。干预之后,ALP水平从200.33降至75.5U/L（p值=0.15）。6名患者中有3名MRC量表得到改善。NSAA保持稳定。没有不良反应。
■这项研究证明了NeuREFIXβ-葡聚糖食品补充剂的安全性及其在2个月的短时间内改善血浆生物标志物和肌肉功能参数的功效。建议通过评估较长持续时间的肌肉功能进行进一步验证，以确认Neu-REFIX食品补充剂作为肌营养不良症的潜在佐剂DMT的功效。
■在这项为期60天的研究中，6例除DMD以外的肌营养不良患者服用了一种8克Neu-REFIXβ-葡聚糖凝胶，以及他们通常的标准治疗方案，以及它们与肌肉功能相关的生物标志物，如血清钙（SC），肌酸磷酸激酶（CPK），和碱性磷酸酶（ALP）水平以及功能改善标准，也就是说，医学研究委员会（MRC）量表和北极星门诊评估（NSAA），在基线和干预后进行评估。
■干预后，SC水平从平均基线值9.28mg/dL显著降低至8.31mg/dL(p值=0.02).p值为0.29时，CPK的平均值从2192.33IU/L降至1567.5IU/L。干预之后,ALP水平从200.33降至75.5U/L（p值=0.15）。6名患者中有3名MRC量表得到改善。NSAA保持稳定。没有不良反应。
■这项研究证明了NeuREFIXβ-葡聚糖食品补充剂的安全性及其在2个月的短时间内改善血浆生物标志物和肌肉功能参数的功效。建议通过评估较长持续时间的肌肉功能进行进一步验证，以确认Neu-REFIX食品补充剂作为肌营养不良症的潜在佐剂DMT的功效。
