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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

PubMedResearch square2026-08-01

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

Ambaw Yohannes A YA, Nana Alissa L AL, Li Zhuoning Z, 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's disease, 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 42539062
阅读全文 →
PubMedBrain : a journal of neurology2026-07-29

Revisiting multifocal motor neuropathy: mechanisms, diagnosis and future directions.

Dubuisson Nicolas J NJ, Rajabally Yusuf A YA, Philips Clive C, Harbo Thomas T et al.

Multifocal Motor Neuropathy (MMN) is an immune-mediated, pure motor neuropathy characterized by slowly progressive, asymmetric limb weakness. It predominantly affects men in mid-adulthood and often presents with distal upper-limb weakness, such as wrist or finger drop. Although the disease course is typically chronic, slowly and sometimes stepwise progressive, timely recognition is critical, as early immunotherapy can prevent irreversible axonal loss and long-term disability. This review aims to provide a comprehensive and clinically integrated overview of MMN, spanning pathogenesis, clinical presentation, diagnostic challenges, and current and emerging therapeutic strategies. Emphasis is placed on translating mechanistic insights into practical diagnostic and treatment frameworks, and on identifying priorities for future research. Pathophysiologically, MMN is best understood as an antibody-mediated nodo-paranodopathy. IgM antibodies directed against GM1 gangliosides bind to GM1-enriched domains at the nodes of Ranvier, activate the classical complement cascade, and disrupt sodium channel clustering and axo-glial integrity. The resulting conduction block constitutes the electrophysiological hallmark of the disease and may initially be reversible. Persistent immune-mediated injury, however, can lead to secondary axonal degeneration and fixed disability. While GM1 IgM antibodies are detected in a substantial proportion of patients, seronegativity does not exclude MMN, potentially reflecting mechanistic heterogeneity and/or assay limitations. Diagnosis relies on the combination of a characteristic clinical phenotype and electrophysiological evidence of motor conduction block with preserved sensory conduction. However, conduction block can be difficult to demonstrate because of proximal lesions, temporal dispersion, or superimposed axonal loss, contributing to diagnostic uncertainty. High-resolution nerve ultrasound and magnetic resonance neurography offer valuable complementary information by revealing focal nerve enlargement or plexus abnormalities, though neither technique is independently diagnostic. Intravenous immunoglobulin (IVIg) remains the established standard of care and the only approved therapy, producing rapid but often transient improvement that necessitates individualized maintenance regimens. Subcutaneous immunoglobulin provides an effective long-term alternative for many patients. Conventional immunosuppressants have shown limited efficacy, whereas emerging complement inhibitors targeting upstream components of the classical pathway represent a promising, mechanism-based strategy currently under clinical evaluation. Despite advances, significant gaps remain. Reliable biomarkers to predict treatment response, monitor disease activity, and detect early axonal injury are lacking. Future progress will require integrated translational models, harmonized outcome measures, and well-designed clinical trials to move toward more durable and personalized therapeutic approaches in MMN.

PMID 42522566
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PubMedInternational journal of molecular sciences2026-07-28

Anti-Alpha-Gal Antibodies Against Gangliosides: Preliminary Data on a New Autoimmune Target in Alzheimer's Disease Patients.

Naso Filippo F, Gandaglia Alessandro A, Sturaro Giulio G, Lepore Alessio A et al.

Human anti-αGal antibodies (Abs), known for their marked polyreactivity, have been detected bound to the gray matter of the brains of Alzheimer's disease (AD) patients, although their targets were unclear. Since αGal is a strictly xenogenic antigen absent in humans, this observation raised questions regarding the nature of the structures recognized by these antibodies. In this study, we investigated their potential interaction with gangliosides-glycan structures that are highly abundant in the central nervous system. Using a competitive inhibition ELISA, serum profiles of anti-αGal Abs isotypes and their indirect cross-reactivity with selected soluble gangliosides were analyzed in AD patients and healthy subjects (HSs). AD patients showed reduced levels of anti-αGal IgG and IgM, but increased IgA compared to HSs. Notably, pre-incubation with GM1, GM2, or GD1b did not reduce αGal-HSA binding in HS sera. In contrast, in AD sera, pre-incubation with GD1b reduced residual αGal-HSA binding for all antibody isotypes; additionally, GM1 inhibited IgM binding, and GM2 inhibited IgA binding. These results should therefore be interpreted as competitive inhibition patterns consistent with ganglioside-associated cross-reactivity rather than as direct evidence of antibody binding to immobilized gangliosides. Overall, the findings provide preliminary evidence that, in AD sera, a fraction of αGal-HSA-reactive antibodies can be competitively inhibited by selected gangliosides. This observation supports the presence of an altered humoral anti-carbohydrate signature in AD and identifies neuronal gangliosides as plausible candidate autologous targets that may help explain the previously reported binding of anti-αGal Abs to gray matter. However, given the indirect nature of the assay, these data should be considered hypothesis-generating and require confirmation by direct binding approaches.

PMID 42511534
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PubMedBiomedicines2026-07-28

Congenital Disorders of Glycosphingolipid Biosynthesis: Ultrarare Severe Syndromes or Relatively Frequent Mild Neurocognitive Illnesses?

Montavoci Linda L, Dei Cas Michele M, Penati Sara S, Trinchera Marco M

Glycosphingolipids (GSLs) are glycoconjugates in which a short and heterogeneous saccharide chain is attached to a lipid moiety called ceramide. Based on their sugar backbone, mammalian GSLs are primarily grouped into the ganglio-, lacto-/neolacto-, and globo-series. Sialic acid-containing GSLs are known as gangliosides. Complex ganglio-series gangliosides are particularly abundant in the brain, whereas simple ganglio-series gangliosides, as well as those belonging to other series or neutral GSLs, are less abundant and typical of non-neural tissues. Congenital disorders in the biosynthesis of the lipid moiety of sphingolipids (SLs) result from defects in enzymes and proteins involved in ceramide biosynthesis and transport. Congenital disorders in the biosynthesis of the sugar chain of GSLs specifically affect ganglio-series ganglioside biosynthesis and are caused by pathogenic variants in GM3 synthase (ST3GAL5) or GM2/GD2/asialo-GM2 synthase (B4GALNT1). Defective variants of the sialyltransferase ST3GAL3 and the galactosyltransferase B4GALT5 have been reported and proposed to impair GSL biosynthesis. The occurrence of these syndromes has provided new insights into the physiological and pathological roles of GSLs. Most of these disorders are associated with completely inactive enzyme variants, leading to severe neurological syndromes. Only a few cases highlighted variants that retained partial activity, resulting in milder phenotypes, which included non-syndromic intellectual disability. It is therefore conceivable that many undiagnosed patients, with mild neurological symptoms, may carry variants retaining residual enzyme activity, insufficient to ensure normal levels of brain GSLs. The purpose of this article is to encourage clinicians to look for additional GLS hereditary disorders associated with a milder phenotype. We also hope to boost future investigations by highlighting the most critical issues emerging from recent literature on SL and GSL biosynthesis and their related defects.

PMID 42511980
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PubMedDiagnostics (Basel, Switzerland)2026-07-28

Multi-Omics Profiling in a Symptomatic Cohort Identifies Coordinated Biomarker Signatures in Ovarian Cancer Serum.

Culp-Hill Rachel R, Nichols Charles M CM, Kilkenny Shannon S, Goldberg Mattie M et al.

Background/Objectives: Ovarian cancer (OC) is a leading cause of cancer-related mortality in women, largely driven by late-stage diagnosis. Five-year survival is just 30% for advanced-stage (III-IV) disease but exceeds 90% for early-stage disease, underscoring the critical need for effective early detection tools. Current standard-of-care biomarkers show limited sensitivity for early-stage OC and lack specificity in symptomatic populations. Most biomarker studies in OC serum evaluate single molecular classes or compare OC to healthy controls, limiting understanding of coordinated biological alterations in circulating proteins, lipids, and metabolites in clinically relevant populations. Methods: We performed integrated multi-omics profiling of serum from a retrospective, case-control cohort of women presenting with vague abdominal symptoms (VAS), including early- and late-stage OC, borderline tumors, benign gynecologic conditions including adnexal masses, GI disorders, and healthy controls. Protein biomarkers were quantified by ELISA, lipidomic profiling was performed by untargeted LC-MS, and ganglioside and metabolomic profiling were performed by semi-targeted LC-MS with metabolite annotation performed against a curated reference library. Results: Consistent with known limitations for early-stage OC detection, CA125 and HE4 levels overlapped substantially with benign gynecologic conditions. Additional proteins also showed limited separation in their expression between early-stage OC and symptomatic controls. In contrast, OC showed unique lipid and metabolite profiles: phospholipids and glycerolipids were decreased, and sphingolipid composition was altered. Borderline and benign conditions exhibited lipid profiles that fall between healthy and OC groups, suggesting a continuum of metabolic changes rather than distinct states between OC and non-OC controls. Sphingolipid alterations included changes in ceramides and sphingomyelins, along with broader dysregulation of ganglioside profiles, including an elevated GD2;O2-to-GD1;O2 ratio. Metabolic profiling showed decreased amino acids and enriched cysteine metabolism in OC, consistent with altered redox balance, along with changes in fatty acids and acyl-carnitines, suggesting altered lipid metabolism and inflammatory mechanisms. Lower levels of glycolytic and TCA cycle intermediates in OC suggested altered mitochondrial metabolism and energetic reprogramming. Pairwise comparisons revealed a gradient of significance between groups, with differences between OC and healthy controls across lipid classes (LPC, PC, PE, TG, SM), gangliosides (GD1, GD2, GD2/GD1 ratio), and metabolites (amino acids, Cys/CySS, TCA cycle); borderlines occupied an intermediate space. Integration of these datasets revealed coordinated cross-omics relationships, identifying links between metabolite, lipid, and protein features. Together, these connections highlight structured, system-level alterations related to lipid remodeling, redox balance, immune signaling, and energy metabolism that no single modality would have revealed in isolation. Conclusions: This study presents an integrated analysis of the lipidome, gangliosome, metabolome, and protein biomarkers within a single clinically relevant symptomatic cohort enriched with multiple stages and subtypes of OC. This multi-omics framework demonstrates that molecular alterations in OC are biologically interconnected across molecular classes. While these findings are discovery-based and require independent validation prior to clinical application, they support the development of clinically deployable multi-omics biomarker strategies for early detection and potential pathways for therapeutic intervention.

PMID 42510006
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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
阅读全文 →

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