Drug Database
GA

gangliosides (Nervomax / gangliosides, Fidia / Neurosido)

✓ Approved

Gramon · therapeutic agent

What is gangliosides?

gangliosides is a therapeutic agent developed by Gramon. It is approved for therapeutic indications via unknown.

Drug Profile

Brand NamesNervomax, gangliosides, Fidia, Neurosido
CompanyGramon
RouteUnknown
StatusApproved
Sources: ClinicalTrials.gov, Gramon

Therapeutic Indications

gangliosides is developed for 4 unique indications across 3 therapeutic areas.

Therapeutic AreaConditionPhase
Nervous system disordersDiabetic neuropathy✓ Approved
Nervous system disordersNeuropathy peripheral✓ Approved
Blood and lymphatic system disordersNeutropenia✓ Approved
Congenital, familial and genetic disordersRetinitis pigmentosa✓ Approved

Related Research Articles

PubMedImmunity, inflammation and disease2026-11-05

Dizziness, Scanning Speech, and Transient Hyperreflexia as Atypical Initial Manifestations of Antiganglioside Q1b-Negative Miller Fisher Syndrome: A Case Report and Literature Review.

Wu Wenjing W, Mao Mengying M, Xu Bin B, Xu Liang'e L

Miller-Fisher syndrome (MFS) is characterized by the triad of ataxia, areflexia, and ophthalmoplegia. To our knowledge, the combination of initial dizziness, scanning speech, transient hyperreflexia with a Babinski sign, and persistent anti-GQ1b seronegativity has not been previously reported. A 27-year-old Chinese Han male patient presented with prominent dizziness and scanning speech as the primary symptoms. Physical examination revealed hyperreflexia of all tendon reflexes initially, along with intention tremor and a positive Babinski's sign on the right side. Subsequently, he exhibited the classic triad of MFS, and cerebrospinal fluid analysis showed albuminocytological dissociation. He was ultimately diagnosed with MFS. Electromyography (EMG) revealed significantly abnormal blink reflexes, dysfunction of the brainstem auditory pathway, particularly within the central nervous system, and a descending sensory nerve action potential (SNAP) in the left sural nerve. Cerebral Magnetic Resonance Imaging (MRI) and ganglioside Q1b (GQ1b) antibodies were both negative. After treatment with intravenous immunoglobulin (IVIG) for 5 days, the patient's condition significantly improved. By the 1-month follow-up, his neurological function had completely recovered. The involvement of dizziness, scanning speech, hyperreflexia, and Babinski's sign in anti-GQ1b-negative MFS is quite rare, indicating early central nervous system (CNS) involvement. The pathogenesis of MFS may have a broader immunological basis rather than being solely attributable to a single autoantibody-mediated response against gangliosides.

PMID 42985973
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PubMedFrontiers in microbiology2026-11-03

CjejuniTyper: integrated in silico typing of Campylobacter jejuni capsule and lipooligosaccharide loci with GBS-associated genomic profiling.

Jurić Dragan D, Ferenčak Ivana I, Grginić Ana Gverić AG, Žmak Ljiljana L et al.

Campylobacter jejuni (C. jejuni) is the leading bacterial cause of gastroenteritis worldwide and the most common antecedent pathogen linked with Guillain-Barré syndrome (GBS), an acute autoimmune neuropathy. The risk of GBS development is associated with the structure of the bacterial lipooligosaccharide (LOS), especially sialylated LOS classes that mimic human peripheral nerve gangliosides, as well as with the Penner heat-stable (HS) capsular polysaccharide (CPS) serotype. Accurate simultaneous determination of LOS class and CPS type is important for surveillance of GBS-associated C. jejuni lineages but currently requires culture-based serology or separate molecular assays. CjejuniTyper is an automated bioinformatic workflow that performs Penner CPS complex prediction, LOS class assignment, Cst-II allele calling, putative ganglioside-mimic prediction, virulence-factor detection, and antimicrobial-resistance profiling from genome assemblies. Penner prediction uses marker-gene searches, full-locus alignment, and subtype-resolution rules, whereas LOS classes are assigned using a BLAST-based scoring and confidence system. The workflow was evaluated using 617 assemblies with previously assigned LOS classes, 96 C. jejuni assemblies with Penner reference labels, and 113 retained genomes comprising 89 isolates previously reported from GBS patients and 24 enteritis controls. LOS typing correctly classified 595 of 617 assemblies, corresponding to 96.4% accuracy, Cohen's kappa of 0.958 and a Matthews correlation coefficient of 0.958. Penner typing correctly classified 93 of 96 assemblies, corresponding to 96.9% accuracy, Cohen's kappa of 0.961, and a Matthews correlation coefficient of 0.962. Most incorrect classifications involved closely related or structurally overlapping loci in fragmented draft assemblies. Among 84 GBS-associated genomes with published LOS-class assignments, CjejuniTyper produced 83 concordant calls, corresponding to 98.8% concordance. In the Bangladesh case-control cohort, HIGH or MODERATE genomic profiles were identified in 34 of 38 GBS-associated isolates and 10 of 24 enteritis controls, corresponding to 89.5% sensitivity, 58.3% specificity, and 73.9% balanced accuracy. Across all 89 retained GBS-associated genomes, 81 (91.0%) received HIGH or MODERATE profiles. These profiles were enriched among GBS-associated isolates but were also present in some controls, indicating genomic mimicry-associated potential rather than patient-level clinical prediction. CjejuniTyper provides a reproducible workflow for integrated C. jejuni capsule and LOS typing with ganglioside-mimic, virulence, and antimicrobial-resistance annotations in a single output.

PMID 42973885
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PubMedBiology open2026-11-02

Implication of N-glycosylations on B4GALNT1 enzyme in its molecular assembly and function.

Ohmi Yuhsuke Y, Koyanagi Hiroka H, Ito Takako T, Imao Yuka Y et al.

B4GALNT1 is a glycosyltransferase essential for the synthesis of complex gangliosides and plays important roles in the nervous system. Although N-glycosylation is thought to regulate B4GALNT1, its functional significance has remained unclear. In this study, we generated human B4GALNT1 mutants carrying single or combined substitutions at three N-glycosylation sites (N1-N3) and analyzed their enzymatic activity, intracellular localization, and molecular assembly using transfected B78 mouse melanoma cells. Loss of N-glycosylation reduced GM2 expression and enzymatic activity in a site-dependent manner, with multiple-site mutants showing greater impairment. Golgi localization was also progressively disrupted as the number of mutated N-glycosylation sites increased. Non-reducing immunoblotting demonstrated that loss of the N1 glycosylation site markedly impaired B4GALNT1 dimer formation, with additional defects caused by the combined loss of the N3 and N2 sites. Predicted dimeric B4GALNT1 structures generated by AlphaFold2 revealed the spatial arrangement of N-glycans, suggesting a dominant role for the N1 site and a supportive role for the N3 site in stabilizing intermolecular contacts. These results indicated that N-glycosylation sites in the three-dimensional structure of B4GALNT1 are critical for the molecular integrity and enzymatic activity.

PMID 42969170
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PubMedInternational journal of molecular sciences2026-10-27

Gangliosides in Cutaneous Melanoma: Molecular Mechanisms, Translational Evidence Gaps, and Preclinical Natural-Compound Leads.

Koo Deog-Bon DB, Kim Kyongtae K, Kim Juhwan J, Yun Hyo Gyeong HG et al.

Cutaneous melanoma remains lethal once metastatic despite advances in immune checkpoint blockade and BRAF/MEK-directed therapy. Gangliosides are surface-accessible sialylated glycosphingolipids that regulate membrane organization, adhesion, and signaling, representing potential biomarkers and therapeutic targets. This narrative review evaluated the translational readiness of ganglioside-directed strategies in cutaneous melanoma, without assuming that evidence from other GD2- or GD3-positive cancers is directly transferable. We synthesized melanoma-specific evidence on GD3-linked focal adhesion signaling, p130Cas/paxillin activity, PI3K-AKT and MAPK outputs, and distinct phenotypes associated with GD3, GD2, and GM3. We then applied an evidence-gated framework distinguishing melanoma clinical evidence, melanoma preclinical evidence, cross-tumor translational evidence, and hypothesis-generating claims. Direct contemporary clinical evidence in melanoma remains limited. Antibody, cellular, and biosynthetic enzyme strategies require diagnostic-quality antigen profiling, demonstration of surface accessibility and functional dependency, and prospective monitoring for antigen escape and neurotoxicity. Natural compounds, particularly quercetin, are mechanistic probes or formulation-enabled leads: a 2024 study reported reduced GM3/GD3 abundance and FAK/paxillin/AKT signaling in melanoma cell lines, but high in vitro concentrations and the lack of pharmacokinetic or in vivo validation preclude therapeutic conclusions. We proposed a minimum evidence package and go/no-go criteria for clinical translation, specifying experiments needed to advance ganglioside biology into biomarker-defined melanoma interventions.

PMID 42941795
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PubMedJournal of colloid and interface science2026-10-27

Tuning membrane domain structure and surface charge through the interplay of lipid composition, ions, and the charge state of ganglioside GM1.

Grava Miriam M, Rondelli Valeria V, Schneck Emanuel E

Gangliosides contribute to membrane organization and are key components of functional membrane domains. Among them, GM1 is particularly relevant because of its preferential association with cholesterol-rich ordered domains and its involvement in membrane-mediated recognition processes. Yet, how its structural and electrostatic properties depend on the local lipid and ionic environment remains poorly understood. Here, we combine grazing-incidence X-ray scattering (GIXS), grazing-incidence X-ray diffraction (GIXD), and total-reflection X-ray fluorescence (TRXF) to investigate GM1-containing lipid monolayers with different phospholipid matrices with or without cholesterol. We show that membrane composition strongly determines the structural response to the ionic environment: cholesterol stabilizes a liquid-ordered state with comparatively weak structural sensitivity to calcium, whereas GM1 and millimolar concentrations of Ca2+ promote denser packing in cholesterol-free lipid mixtures and shift the phase behavior toward ordered states. Importantly, quantification of interfacial ion adsorption reveals that GM1 deprotonation and the resulting membrane charge are coupled to molecular packing. These results establish membrane organization and interfacial charge regulation as mutually coupled properties, controlled by the interplay between lipid composition, ion accumulation, and the charge state of GM1, with potential implications for molecular interactions at biomembrane surfaces.

PMID 42945675
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PubMedMolecules (Basel, Switzerland)2026-10-27

The Disordome Paradigm: How Intrinsic Disorder Encodes Emergent Biological Function.

Fantini Jacques J, Lefebvre Marine M, Aulas Anaïs A, Chahinian Henri H et al.

Classical structural biology equates function with a fixed three-dimensional fold, a view challenged by intrinsically disordered proteins (IDPs) and regions (IDRs), which populate broad conformational ensembles rather than a unique structure. This article introduces the "disordome" paradigm, extending Uversky's "unfoldome" to describe how function emerges from electrostatic and hydration landscapes that reshape reversibly upon encounter with a partner. Using α-synuclein binding to ganglioside-enriched rafts and IDR-driven liquid-liquid phase separation, we argue that transient folding-upon-binding events, potentially entropically cheap, generate information-rich complexes and condensates that dissolve once conditions change, consistent with the second law of thermodynamics. We trace this logic to overlooked precursors: in 1957 Klotz invoked rubber elasticity to introduce entropy as the driver of protein "disorientation", while in 1962 Bernal described such proteins as chains "coiled in different ways" and formulated a "two-atom rule" whereby bonding constraints fix atomic geometry only up to two atoms apart. We argue that the local under-determination described by this two-atom rule, left unresolved in IDPs by any conserved network of contacts, helps explain why AlphaFold fails on IDPs. A quantum-mechanical pillar (dispersion, OH-π contacts, tunneling) is proposed as underrepresented in IDP research. We discuss implications for the origin of life, cellular regulation via condensates, and a therapeutic logic ("target the target, not the arrow") illustrated by AmyP53, which occupies raft gangliosides rather than disordered amyloid proteins in Alzheimer's and Parkinson's diseases.

PMID 42943733
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