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gangliosides (Nervomax / gangliosides, Fidia / Neurosido)

✓ Approved

Gramon · 治疗药物

什么是 gangliosides？

gangliosides 是一种治疗药物，由Gramon研发。该药已获批，用于治疗相关适应症，给药途径：Unknown。

药物档案

商品名Nervomax, gangliosides, Fidia, Neurosido
公司Gramon
给药途径Unknown
状态Approved
来源： ClinicalTrials.gov, Gramon

治疗适应症

gangliosides 针对 4 个适应症，涉及 3 个治疗领域。

治疗领域疾病/病症分期
Nervous system disordersDiabetic neuropathy✓ Approved
Nervous system disordersNeuropathy peripheral✓ Approved
Blood and lymphatic system disordersNeutropenia✓ Approved
Congenital, familial and genetic disordersRetinitis pigmentosa✓ Approved

相关研究文献

PubMedbioRxiv : the preprint server for biology2026-07-17

Shared lipidome and proteome signatures of frontotemporal lobar degeneration and Alzheimer's disease.

Ambaw Yohannes Y, Nana Alissa A, Zhuoning Li L, Singh Shubham S et al.

Frontotemporal lobar degeneration (FTLD) and Alzheimer's disease (AD) differ in their clinical features and genetic etiologies but share progressive cognitive decline. Emerging evidence implicates lipid dysregulation in neurodegeneration, but its extent across FTLD subtypes and how it compares to AD are unclear. Here, we performed integrated lipidomic and proteomic analyses of matched frontal (disease-vulnerable) and occipital (relatively spared) post-mortem cortices from individuals with genetic and sporadic FTLD-TDP, FTLD-tau (Pick disease's, PiD), AD, and controls. FTLD and AD exhibited convergent lipid alterations, including reduced levels of cardiolipins and phosphatidylethanolamines, alongside increased gangliosides, diacylglycerols, cholesterol esters, acylcarnitines, and coenzyme Q, with generally greater changes in FTLD frontal cortex. FTLD displayed additional alterations, including reductions in bis(monoacylglycerol)phosphate, ceramides, phosphatidylserines, phosphatidylinositols, and sulfatides. These lipid changes were accompanied by proteomic alterations involving lysosomal proteins, phospholipases, phospholipid remodeling enzymes, and fatty acid oxidation pathways. Although lipidomic and proteomic signatures were broadly shared across FTLD subtypes, GRN associated FTLD-TDP and PiD showed the most extensive alterations. Triglycerides were selectively reduced in PiD in association with decreased DGAT1 expression, whereas cholesterol esters were elevated across all subtypes except C9orf72 associated FTLD-TDP. These findings identify shared disruptions in lipid homeostasis and lysosomal lipid metabolism across FTLD and AD, highlighting convergent metabolic pathways underlying neurodegeneration.

PMID 42465421
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PubMedThe Journal of neuroscience : the official journal of the Society for Neuroscience2026-07-17

Transcription co-repressors Panky and Panky-like modulate ganglioside levels and are essential for retinal cone photoreceptor structure and survival.

Tu Hung-Ya HY, Gyoten Daichi D, Sumihiro Hitoshi H, Michikawa Kayoko K et al.

The biological functions and mechanisms of many cell-type-specific transcriptional cofactors remain unclear. We previously identified Panky (Ankrd33) as a repressive transcriptional co-factor that suppresses Crx-mediated transactivation of photoreceptor genes; however, the biological function of Panky remains unclarified. Here, we investigated the functions of Panky and its paralog, Panky-like (Ankrd33b), in mice of either sex. Single knockout (KO) mouse retinas of Panky or Panky-like did not show significant alterations at both histological and functional levels compared to those of control mice. We then established and examined Panky and Panky-like double knockout (PPL DKO) mice. PPL DKO mice exhibited decreased light-evoked activities detected by electroretinogram (ERG), impaired cone photoreceptor morphology revealed by immunohistochemistry, and structural deformity of cone outer segments and synaptic terminals indicated by transmission electron microscopy (TEM), followed by cone death at a later stage. Lipidomics analysis revealed elevated ganglioside levels in PPL DKO retinas, consistent with the increased immunoreactivity of GT1b and GD3 in the outer segment layer. RNA-seq analysis was conducted in a cone-enriched context resulting from Nrl deficiency and showed that the Cerkl gene, whose mutations in humans cause retinitis pigmentosa (RP26), was upregulated in Panky/Panky-like/Nrl triple KO mice. Transcriptional assays showed that PPL suppressed Crx-mediated transactivation of Cerkl In addition, AAV-mediated Cerkl overexpression in the mouse retina induced significant photoreceptor cell death. Taken together, these findings suggest that the repressive Panky and Panky-like co-factors, which modulate Crx transcriptional activities and thus ganglioside levels in the mouse retina, are essential for cone photoreceptor structure and maintenance.Significance statement The retina is a light-sensing neural tissue in the eye. Photoreceptor cells (PRs) in the retina receive light stimuli. Rod PRs mediate dim-light vision, whereas cone PRs are responsible for bright and color vision. The present study identified that Panky and Panky-like genes are predominantly expressed in PRs in the mouse retina. Panky and Panky-like double-knockout mice exhibit impaired cone cell morphology, cone cell death, and visual dysfunction. In addition, gangliosides, which are chemically bonded lipids and sugars essential for cell membrane stability and function, accumulate in the photoreceptor layer. This study showed that Panky and Panky-like genes regulate ganglioside levels in the retina and play essential roles in the structure and maintenance of cone photoreceptor cells.

PMID 42463452
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PubMedThe Journal of reproduction and development2026-07-16

B3GALT4-dependent gangliosides are required for normal late spermatogenesis and male fertility.

Nagata Shino S, Nishioka Saki S, Ikawa Masahito M, Ozawa Manabu M

Gangliosides play important roles in membrane organization and cellular signaling. Although their involvement in spermatogenesis has been suggested, the specific functions of individual ganglioside species during each stage of germ cell development remain poorly understood. In this study, we generated beta-1,3-galactosyltransferase 4(B3galt4) knockout (KO) mice to investigate the role of B3GALT4-dependent ganglioside in male germ cell development. Immunostaining analyses demonstrated that B3galt4 deficiency caused selective alterations in ganglioside distribution. Although testis size did not differ significantly between KO and control mice, KO males were completely infertile. Histological examination revealed that spermatogenesis in KO mice proceeded to the elongating spermatid stage; however, abnormalities were observed during the late stages of spermatogenesis, including a reduced number of elongating/elongated spermatids and multinucleated giant cells in subsets of seminiferous tubules. Consistent with these observations, the number of sperm recovered from the cauda epididymis was markedly decreased, accompanied by morphological abnormalities and impaired motility. Collectively, these findings demonstrate that B3galt4 deficiency compromises late spermatogenesis and sperm function, resulting in male infertility. Our results further suggest that B3GALT4-dependent gangliosides have stage-dependent roles during late spermatogenesis and sperm development.

PMID 42457561
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PubMedFrontiers in neurology2026-07-11

Gangliosides in the 21st century: therapeutic prospects for the brain and spine.

Magistretti Pierre J PJ, Finsterwald Charles C, Itokazu Yutaka Y, Sipione Simonetta S et al.

Gangliosides are sialylated glycosphingolipids highly enriched in the central nervous system, where they regulate membrane signaling, metabolism, neurogenesis, and immune responses. This Review integrates recent advances across distinct experimental and clinical domains. First, recent studies demonstrate that the monosialoganglioside GM1 enhances astrocyte-neuron metabolic coupling via the astrocyte-neuron lactate shuttle, thereby supporting neuronal bioenergetics and resilience. Complementary mechanistic work shows that specific gangliosides regulate adult neurogenesis through developmentally controlled epigenetic and transcriptional programs. In Huntington's disease models, preclinical evidence indicates that GM1 and related gangliosides attenuate microglia-mediated inflammatory responses and promote proteostasis through extracellular vesicle-dependent clearance of misfolded proteins. Finally, clinical evidence from acute spinal cord injury demonstrates that GM1 administration accelerates neurological recovery, underscoring its translational relevance. Together, these findings position gangliosides as multi-target modulators of neural repair and inflammation, and highlight their potential for therapeutic development.

PMID 42434075
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PubMedACS infectious diseases2026-07-07

Influenza A Virus Binding to α,2-3- and α,2-8-Linked Sialo-Gangliosides Reconstituted in Phase-Separated Vesicles.

Dhanawat Garvita G, Dey Manorama M, Agrawal Shriya S, Lodha Kunika K et al.

We investigated the binding of influenza A viruses (IAVs) to gangliosides containing either α,2-3- or α,2-8-linked sialic acid residues, or a combination of both. The respective gangliosides were reconstituted into phase-separated giant unilamellar vesicles to mimic the cellular membrane environment. Dual-color fluorescence imaging of influenza particles interacting with these vesicles revealed that the order of virus binding density and the lateral mobility of bound particles does not correlate with (i) the intrinsic affinity of viral hemagglutinin (HA) for specific sialic acid conformations or (ii) the number of sialic acid residues present in the gangliosides. Instead, we observe that the extent of viral attachment closely follows the surface charge of the ganglioside-rich vesicles. Because influenza particles carry a net negative charge, they experience electrostatic repulsion when approaching negatively charged membranes. Consequently, electrostatic interactions modulate the ability of viruses to establish multivalent contacts with membrane-bound sialic acids. Taken together, our findings demonstrate that electrostatic repulsion, glycosidic linkage, and lipid packing collectively govern influenza binding to phase-separated membranes. These results suggest that the commonly discussed preferential binding of influenza to specific sialic acid linkages and its association with viral species tropism should be reconsidered in the broader context of membrane surface charge and lipid organization.

PMID 42409417
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PubMedAnalytical chemistry2026-07-06

Comprehensive Isomer-Resolved Ganglioside Profiling by HILIC-nanoESI-MS/MS.

Troppmair Nina N, Santol Jonas J, Baumert Mark M, Opálka Lukáš L et al.

Gangliosides are structurally complex glycosphingolipids that regulate cellular signaling via interactions with extracellular binding partners and by influencing protein function within the membrane. Their comprehensive analysis remains analytically challenging, in part due to low endogenous abundance, extensive structural diversity, and the frequent occurrence of isomeric species arising from both glycan and ceramide moieties. Here, we present a hydrophilic interaction liquid chromatography (HILIC) nanoelectrospray ionization tandem mass spectrometry (nanoESI-MS/MS) method specifically designed to achieve isomer-resolved analysis and molecular species-level quantification of gangliosides. To improve characterization of low-abundance fatty acyl fragments, the workflow was extended by combining nanoESI with online fraction collection and subsequent direct infusion. The method demonstrated stable chromatographic performance, high sensitivity, and reproducible quantitative results across ten mouse tissues. Application of the workflow enabled quantification of 80 ganglioside molecular species belonging to 18 subclasses and revealed pronounced tissue-specific differences in subclass distribution and ceramide composition. Overall, this study establishes a robust and sensitive platform for comprehensive ganglioside analysis, providing new opportunities to investigate ganglioside diversity, regulation, and function in complex biological systems.

PMID 42406901
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