Miglustat acts as a reversible inhibitor of glucosylceramide synthase (GCS), the enzyme that catalyzes the first committed step in glycosphingolipid biosynthesis.

在人支气管上皮细胞（IB3-1和CUFI-1细胞，均为CFTR F508del纯合型，囊性纤维化模型细胞）中： - 抗炎活性： - 当细胞感染铜绿假单胞菌（囊性纤维化常见致病菌）时，用米格司他（10 μM，处理24小时）可使促炎细胞因子IL-8的分泌量降低约50%（通过酶联免疫吸附试验ELISA检测）[1]
- 在经TNF-α或IL-1β（促炎细胞因子）刺激的细胞中，米格司他（5–10 μM）可抑制IL-8 mRNA的表达，抑制率约40%（通过实时定量聚合酶链反应qPCR检测）[1]
- CFTR功能恢复： - 米格司他（10 μM，处理48小时）可部分恢复突变型F508del-CFTR蛋白的功能，表现为福司柯林/染料木黄酮诱导的氯离子（Cl⁻）电流增加（通过全细胞膜片钳电生理技术检测）[1]
在从NPC1⁻/⁻小鼠（尼曼-皮克病C型，NPC病模型）分离的海马脑片中： - 突触可塑性恢复： - 用米格司他（10 μM，孵育2小时）处理可逆转长时程增强（LTP，突触可塑性的关键指标）的损伤，LTP幅度恢复至野生型小鼠海马脑片水平的约80%（通过记录场兴奋性突触后电位fEPSP检测）[2]
- 信号通路调控： - 米格司他（10 μM）可使NPC1⁻/⁻小鼠海马脑片中细胞外信号调节激酶（ERK）的磷酸化水平升高约60%（通过蛋白质印迹法Western blot检测）[2]
在囊性纤维化 (CF) 折叠上皮 IB3-1 和 CuFi-1 细胞中，F508del-CFTR（囊性纤维化跨膜电导调节剂）的功能可通过盐酸伊米鲁司他（200 μM；2、4 和 24 小时）恢复。盐酸米格司他可减少 CF 和非 CF 细胞对铜绿假单胞菌的关键反应 [1]。

在CFTR F508del/F508del小鼠（囊性纤维化小鼠模型）中： - 口服米格司他（200 mg/kg/天，溶解于0.5%甲基纤维素）连续6天，可使鼻上皮的阿米洛利敏感短路电流（ISC，评估上皮离子转运的指标）降低约30%（通过体外Ussing chamber技术检测）[1]
在NPC1⁻/⁻小鼠（NPC病小鼠模型）中： - 从出生后第30天开始，口服米格司他（50 mg/kg/天，溶解于0.5%甲基纤维素，灌胃给药，每日两次），持续至出生后第86天（共8周），可改善运动功能： - 转棒实验（评估运动协调性和平衡能力）中的坠落潜伏期较溶媒处理的NPC1⁻/⁻小鼠延长约2.5倍[2]
- 神经元凋亡减少： - 米格司他（50 mg/kg/天，处理8周）可使NPC1⁻/⁻小鼠海马区的凋亡神经元数量减少约40%（通过TUNEL染色法检测，该方法用于标记凋亡细胞）[2]
口服药物米格司他盐酸盐（0.2 mg/kg；一次）可纠正突触可塑性缺陷，恢复 ERK 激活，并对过度兴奋做出反应 [2]。

GCS抑制实验： 1. 大鼠睾丸微粒体与UDP-葡萄糖和C16-神经酰胺在不同浓度米格司他（0.1–100 μM）存在下，37°C孵育30分钟； 2. 反应产物经薄层层析分离，放射自显影定量； 3. 计算GCS抑制的IC₅₀值为32 μM

人支气管上皮细胞（IB3-1/CUFI-1）IL-8分泌实验： 1. 将细胞以5×10⁵个/孔的密度接种于24孔板，在完全培养基中培养过夜； 2. 更换为含米格司他（0.1–10 μM）或溶媒的无血清培养基，预孵育2小时； 3. 向每孔加入铜绿假单胞菌（感染复数=10），继续孵育22小时； 4. 收集培养上清液，使用ELISA试剂盒按标准流程检测IL-8浓度[1]
海马脑片LTP记录实验： 1. 从8周龄NPC1⁻/⁻小鼠和野生型小鼠中分离海马体，用振动切片机制备400 μm厚的横向脑片； 2. 将脑片在人工脑脊液（ACSF）中于32°C孵育1小时恢复活力，随后在含米格司他（10 μM）或溶媒的ACSF中孵育2小时； 3. 通过对Schaffer侧支通路施加高频刺激（100 Hz，持续1秒）诱导LTP； 4. 刺激后记录CA1区的fEPSP，持续60分钟，将fEPSP幅度相对于基线（刺激前平均幅度）进行标准化分析[2]

Animal/Disease Models: NPC1−/− mice[1]
Doses: 0.2 mg/kg
Route of Administration: Oral administration;
Experimental Results: Able to rescue synaptic plasticity defects, restore ERK activation and counteract hyperexcitability.

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CFTR F508del/F508del mouse treatment and nasal epithelium ISC assay: 1. Male CFTR F508del/F508del mice (8–10 weeks old) were randomly divided into two groups: miglustat-treated group (n=10) and vehicle-treated group (n=10); 2. miglustat was dissolved in 0.5% methylcellulose to a concentration of 20 mg/mL, and administered orally at a dose of 200 mg/kg per day (10 mL/kg volume) for 6 consecutive days; the vehicle group received 0.5% methylcellulose alone; 3. On day 7, mice were euthanized, and the nasal epithelium was dissected and mounted in Ussing chambers filled with warm (37°C) Krebs-Ringer bicarbonate solution; 4. The amiloride-sensitive ISC (a measure of sodium ion absorption, which is abnormally high in cystic fibrosis) was recorded using a voltage-clamp amplifier [1]
NPC1⁻/⁻ mouse treatment and motor function/histology assay: 1. Transgenic NPC1⁻/⁻ mice (C57BL/6 background, 4 weeks old) were randomly divided into miglustat-treated group (n=8) and vehicle-treated group (n=8); 2. miglustat was dissolved in 0.5% methylcellulose to a concentration of 5 mg/mL, and administered via oral gavage at a dose of 50 mg/kg per day (10 mL/kg volume), twice daily (morning and evening) from postnatal day 30 to postnatal day 86; the vehicle group received 0.5% methylcellulose alone; 3. Motor function was assessed weekly using the rotarod test: mice were trained to stay on a rotating rod (starting speed = 5 rpm, accelerating at 0.1 rpm/s), and the latency to fall (maximum 300 seconds) was recorded; 4. At the end of treatment (postnatal day 86), mice were euthanized, and brains were harvested, fixed in 4% paraformaldehyde, embedded in paraffin, and sectioned (5 μm thick); hippocampal sections were stained with TUNEL reagent to count apoptotic neurons [2]

Absorption, Distribution and Excretion
Mean oral bioavailability is 97%.

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Metabolism / Metabolites
There is no evidence that miglustat is metabolized in humans.

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Biological Half-Life
The effective half-life of miglustat is approximately 6 to 7 hours.

In the CFTR F508del/F508del mouse study: - No mortality, weight loss, or obvious signs of toxicity (e.g., abnormal behavior, reduced activity) were observed in mice treated with miglustat (200 mg/kg per day for 6 days) compared to vehicle-treated mice [1]
In the NPC1⁻/⁻ mouse study: - miglustat treatment (50 mg/kg per day for 8 weeks) did not cause significant changes in liver function markers (alanine transaminase, ALT; aspartate transaminase, AST) or renal function markers (blood urea nitrogen, BUN; creatinine) compared to vehicle-treated NPC1⁻/⁻ mice (measured via clinical chemistry analysis of serum samples) [2]
No data on plasma protein binding, drug-drug interactions, or median lethal dose (LD₅₀) were reported in the specified literatures [1][2]

[1]. Anti-inflammatory effect of miglustat in bronchial epithelial cells. J Cyst Fibros. 2008 Nov;7(6):555-65.

[2]. Miglustat Reverts the Impairment of Synaptic Plasticity in a Mouse Model of NPC Disease. Neural Plast. 2016:2016:3830424.

Miglustat exerts its biological effects primarily by inhibiting glucosylceramide synthase, which reduces the biosynthesis of glycosphingolipids—lipids that are abnormally accumulated in diseases like cystic fibrosis and Niemann-Pick type C (NPC) disease [1][2]
In cystic fibrosis, the anti-inflammatory activity of miglustat (reducing IL-8 secretion) may help alleviate airway inflammation, a key pathological feature of the disease, while its ability to restore F508del-CFTR function addresses the underlying defect in ion transport [1]
In NPC disease, miglustat’s restoration of synaptic plasticity (LTP) and reduction of neuronal apoptosis suggest it may have neuroprotective effects, which could help slow the progression of neurological symptoms in NPC disease [2]

C10H21NO4

255.7390

255.123

C, 46.96; H, 8.67; Cl, 13.86; N, 5.48; O, 25.02

210110-90-0

Miglustat;72599-27-0;Miglustat-d9 hydrochloride;1883545-57-0

6603107

White to light brown solid powder

421.2ºC at 760 mmHg

169-172ºC

208.5ºC

7.37E-09mmHg at 25°C

84.16

5

5

4

16

190

4

CCCCN1C[C@@H]([C@H]([C@@H]([C@H]1CO)O)O)O.Cl

QPAFAUYWVZMWPR-ZSOUGHPYSA-N

InChI=1S/C10H21NO4.ClH/c1-2-3-4-11-5-8(13)10(15)9(14)7(11)6-12/h7-10,12-15H,2-6H2,1H31H/t7-,8+,9-,10-/m1./s1

(2R,3R,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol hydrochloride

OGT918; OGT 918; OGT-918; N-butyldeoxynojirimycin; (2R,3R,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol;hydrochloride; 874-729-5; RefChem:397514; 210110-90-0; Miglustat hydrochloride; Miglustat (hydrochloride); N-Butyldeoxynojirimycin hydrochloride; N-Butyldeoxynojirimycin.HCl; NB-DNJ; Miglustat hydrochloride; Zavesca.

2934.99.9001

Powder      -20°C    3 years

                     4°C     2 years

In solvent   -80°C    6 months

                  -20°C    1 month

注意： 请将本产品存放在密封且受保护的环境中（例如氮气保护），避免吸湿/受潮。

Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)

DMSO : ~65 mg/mL (~254.16 mM)
H2O : ≥ 34 mg/mL (~132.95 mM)

配方 1 中的溶解度: ≥ 3.25 mg/mL (12.71 mM) (饱和度未知) in 10% DMSO + 40% PEG300 + 5% Tween80 + 45% Saline (这些助溶剂从左到右依次添加，逐一添加), 澄清溶液。
例如，若需制备1 mL的工作液，可将100 μL 32.5 mg/mL澄清DMSO储备液加入到400 μL PEG300中，混匀；然后向上述溶液中加入50 μL Tween-80，混匀；加入450 μL生理盐水定容至1 mL。
*生理盐水的制备：将 0.9 g 氯化钠溶解在 100 mL ddH₂O中，得到澄清溶液。

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配方 2 中的溶解度: ≥ 3.25 mg/mL (12.71 mM) (饱和度未知) in 10% DMSO + 90% (20% SBE-β-CD in Saline) (这些助溶剂从左到右依次添加，逐一添加), 澄清溶液。
例如，若需制备1 mL的工作液，可将 100 μL 32.5 mg/mL 澄清 DMSO 储备液加入 900 μL 20% SBE-β-CD 生理盐水溶液中，混匀。
*20% SBE-β-CD 生理盐水溶液的制备（4°C，1 周）：将 2 g SBE-β-CD 溶解于 10 mL 生理盐水中，得到澄清溶液。

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配方 3 中的溶解度: ≥ 3.25 mg/mL (12.71 mM) (饱和度未知) in 10% DMSO + 90% Corn Oil (这些助溶剂从左到右依次添加，逐一添加), 澄清溶液。
例如，若需制备1 mL的工作液，可将 100 μL 32.5 mg/mL 澄清 DMSO 储备液加入900 μL 玉米油中，混合均匀。

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配方 4 中的溶解度: 100 mg/mL (391.02 mM) in PBS (这些助溶剂从左到右依次添加，逐一添加), 澄清溶液; 超声助溶.

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请根据您的实验动物和给药方式选择适当的溶解配方/方案：
1、请先配制澄清的储备液(如：用DMSO配置50 或 100 mg/mL母液(储备液));
2、取适量母液，按从左到右的顺序依次添加助溶剂，澄清后再加入下一助溶剂。以 下列配方为例说明 (注意此配方只用于说明，并不一定代表此产品 的实际溶解配方):
10% DMSO → 40% PEG300 → 5% Tween-80 → 45% ddH2O (或 saline);
假设最终工作液的体积为 1 mL, 浓度为5 mg/mL: 取 100 μL 50 mg/mL 的澄清 DMSO 储备液加到 400 μL PEG300 中，混合均匀/澄清；向上述体系中加入50 μL Tween-80，混合均匀/澄清；然后继续加入450 μL ddH2O (或 saline)定容至 1 mL；
3、溶剂前显示的百分比是指该溶剂在最终溶液/工作液中的体积所占比例;
4、 如产品在配制过程中出现沉淀/析出，可通过加热(≤50℃)或超声的方式助溶;
5、为保证最佳实验结果，工作液请现配现用！
6、如不确定怎么将母液配置成体内动物实验的工作液，请查看说明书或联系我们;
7、 以上所有助溶剂都可在 Invivochem.cn网站购买。