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RARE DISEASE
Guillain-Barré syndrome
Guillain-Barré syndrome
Guillain-Barré syndrome
Synonyms: GBS, Guillain-Barré-Strohl syndrome
Synonyms: GBS, Guillain-Barré-Strohl syndrome
Synonyms: GBS, Guillain-Barré-Strohl syndrome
Drug discovery
11
drugs
With orphan designations
Overview
Guillain-Barré syndrome (GBS) is an acute immune-mediated polyradiculoneuropathy and the most common cause of acute flaccid paralysis worldwide [2][5]. Characterized by ascending paralysis, sensory abnormalities, and autonomic dysfunction, it typically follows infections or vaccinations [5][16]. Diagnosis relies on clinical presentation, cerebrospinal fluid analysis, and nerve conduction studies [6][11]. First-line treatments include IV immunoglobulin (IVIG) and plasma exchange [3][8], with most patients achieving functional recovery despite potential long-term disability [1][16].
Categories: rare neurological diseases
Research Papers
1,562 drug discovery papers about Guillain-Barré syndrome, with 2 first-in-class and 32 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
1,562 drug discovery papers about Guillain-Barré syndrome, with 2 first-in-class and 32 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
2026-07-11 | Impact of Gut Microbiota Dysbiosis in Treatment Outcomes of Guillain-Barré Syndrome.
The gut-microbiota plays a significant role in neuro-autoimmune diseases, like Guillain-Barré syndrome (GBS), a post-infectious disorder of the peripheral nervous system (PNS). By altering host immunity, gut dysbiosis may impact the standard therapeutic efficacy of intravenous immunoglobulin (IVIg) and plasma exchange (PE) and thereby, disease progression in GBS. In this study, we investigated the impact of gut-microbiota diversity on therapeutic outcomes of IVIg and PE in patients with GBS. We enrolled 60 patients with GBS categorized into three treatment groups: IVIg-treated, PE, and supportive-care along with 60 age-sex-matched healthy controls from Bangladesh. Fecal microbial DNA was extracted at different timepoints and sequenced. Clinical and sequenced data were analyzed using the Qiime2-Dada2 pipeline. Gut-microbial diversity significantly differed in patients with GBS before and after treatment (Shannon: p = 0.037; Evenness: p = 0.012), particularly in IVIg-treated patients, showing significant changes after 6-month treatment (Shannon: p = 0.034; Evenness: p = 0.011). Beta diversity indicated microbiota restoration in IVIg-treated patients toward a healthy state after treatment (p ≤ 0.001). Phylum Actinobacteriota (p = 0.026), genera Bifidobacterium (p = 0.006), and Enterococcus (p ≤ 0.0001) were relatively abundant in severe patients. Mechanically ventilated patients showed significant gut-microbial diversity (Unweighted-UniFrac; p ≤ 0.001). Biomarker analysis identified a higher abundance of Eubacterium, Bacteroides, Escherichia-Shigella, and Actinomyces in GBS patients. Our exploratory study indicates that gut microbiota dysbiosis is associated with GBS severity and IVIg-treatment outcomes. IVIg-treatment promoted partial restoration of microbial diversity, suggesting a potential immunomodulatory effect mediated through the microbiota. Further studies using meta-transcriptomics are warranted to define functional consequences of microbial shifts in treatment responses of GBS.
2026-07-08 | Neurological complications of Zika virus and their impact on quality of life: a narrative review.
Zika virus (ZIKV), known for its alarming health consequences, is primarily transmitted by infected Aedes spp. mosquitoes but can also spread through sexual contact and from mother to child during pregnancy. While most infections are mild or asymptomatic, ZIKV has been linked to severe neurological disorders, such as Guillain-Barré syndrome (GBS) in adults and congenital Zika syndrome (CZS) in infants. While the substantial economic burden and the range of these disorders have been described, the potential long-term effects and the impact on quality of life (QoL) remain inadequately understood, presenting challenges for both public health and clinical care. This narrative review describes the broader spectrum of neurological complications associated with ZIKV infection and explores their impact on the QoL of patients and caregivers, aiming to deepen the public and clinical understanding of the topic, which is crucial for developing effective prevention and treatment strategies. Beyond the established links to GBS and CZS, ZIKV has been associated with other neurological complications, such as encephalitis and myelitis, highlighting the complex clinical course of the disease. These neurological outcomes can lead to lifelong disabilities, profoundly affecting patients' and caregivers' QoL. Mothers of children with CZS may experience heightened anxiety, depression, and overall reduced QoL, while many individuals with GBS struggle to regain the QoL they had pre-illness. The limited availability of high-quality data, primarily relying on case reports, highlights the need for more comprehensive research to establish clear causal links and better understand the long-term consequences of ZIKV-related neurological complications. Prioritizing research on long-term effects, management, and prevention strategies is crucial to improving patient outcomes and guiding future clinical care.
2026-07-07 | Arbovirus-Associated Guillain-Barré Syndrome: A Systematic Review and Meta-Analysis of Clinical Characteristics, Subtypes, and Vaccine Associations.
Arboviral infections are increasingly recognized as triggers of Guillain-Barré syndrome (GBS), yet the clinical spectrum, subtype distribution, and strength of association across different arboviruses remain incompletely characterized. This systematic review and meta-analysis evaluated the epidemiology, clinical features, and outcomes of arbovirus-associated GBS. PubMed, Scopus, Web of Science, and Google Scholar were searched through early 2025 following PRISMA 2020 guidelines. Observational studies were included in the quantitative meta-analysis, while case reports and case series were synthesized qualitatively. Random-effects models were used to estimate pooled prevalence, odds ratios, and clinical outcomes. One hundred studies were included, comprising 74 case reports/case series, 19 prevalence studies, and seven case-control studies. The pooled prevalence of GBS among individuals with arboviral infection was 1% (95% CI: 0.3%-3.3%), whereas 37% (95% CI: 22%-54%) of patients with GBS had laboratory evidence of recent arboviral infection. Arboviral co-infections occurred in 16% (95% CI: 8%-31%) of confirmed cases. Case-control studies demonstrated a significant association between Zika virus infection and GBS (OR = 8, 95% CI: 2-34). Qualitative synthesis showed frequent intensive care admission, mechanical ventilation, disability, and mortality. Demyelinating subtypes predominated in Zika virus-associated GBS, whereas axonal variants were more common following Japanese encephalitis virus infection. Multiple arboviruses are associated with GBS, with the strongest evidence for Zika virus. Arbovirus-associated GBS frequently results in severe neurological outcomes, highlighting the need for standardized diagnostics, enhanced surveillance, and prospective multicenter studies to improve understanding of disease mechanisms and optimize patient management.
2026-07-11 | Impact of Gut Microbiota Dysbiosis in Treatment Outcomes of Guillain-Barré Syndrome.
The gut-microbiota plays a significant role in neuro-autoimmune diseases, like Guillain-Barré syndrome (GBS), a post-infectious disorder of the peripheral nervous system (PNS). By altering host immunity, gut dysbiosis may impact the standard therapeutic efficacy of intravenous immunoglobulin (IVIg) and plasma exchange (PE) and thereby, disease progression in GBS. In this study, we investigated the impact of gut-microbiota diversity on therapeutic outcomes of IVIg and PE in patients with GBS. We enrolled 60 patients with GBS categorized into three treatment groups: IVIg-treated, PE, and supportive-care along with 60 age-sex-matched healthy controls from Bangladesh. Fecal microbial DNA was extracted at different timepoints and sequenced. Clinical and sequenced data were analyzed using the Qiime2-Dada2 pipeline. Gut-microbial diversity significantly differed in patients with GBS before and after treatment (Shannon: p = 0.037; Evenness: p = 0.012), particularly in IVIg-treated patients, showing significant changes after 6-month treatment (Shannon: p = 0.034; Evenness: p = 0.011). Beta diversity indicated microbiota restoration in IVIg-treated patients toward a healthy state after treatment (p ≤ 0.001). Phylum Actinobacteriota (p = 0.026), genera Bifidobacterium (p = 0.006), and Enterococcus (p ≤ 0.0001) were relatively abundant in severe patients. Mechanically ventilated patients showed significant gut-microbial diversity (Unweighted-UniFrac; p ≤ 0.001). Biomarker analysis identified a higher abundance of Eubacterium, Bacteroides, Escherichia-Shigella, and Actinomyces in GBS patients. Our exploratory study indicates that gut microbiota dysbiosis is associated with GBS severity and IVIg-treatment outcomes. IVIg-treatment promoted partial restoration of microbial diversity, suggesting a potential immunomodulatory effect mediated through the microbiota. Further studies using meta-transcriptomics are warranted to define functional consequences of microbial shifts in treatment responses of GBS.
2026-07-08 | Neurological complications of Zika virus and their impact on quality of life: a narrative review.
Zika virus (ZIKV), known for its alarming health consequences, is primarily transmitted by infected Aedes spp. mosquitoes but can also spread through sexual contact and from mother to child during pregnancy. While most infections are mild or asymptomatic, ZIKV has been linked to severe neurological disorders, such as Guillain-Barré syndrome (GBS) in adults and congenital Zika syndrome (CZS) in infants. While the substantial economic burden and the range of these disorders have been described, the potential long-term effects and the impact on quality of life (QoL) remain inadequately understood, presenting challenges for both public health and clinical care. This narrative review describes the broader spectrum of neurological complications associated with ZIKV infection and explores their impact on the QoL of patients and caregivers, aiming to deepen the public and clinical understanding of the topic, which is crucial for developing effective prevention and treatment strategies. Beyond the established links to GBS and CZS, ZIKV has been associated with other neurological complications, such as encephalitis and myelitis, highlighting the complex clinical course of the disease. These neurological outcomes can lead to lifelong disabilities, profoundly affecting patients' and caregivers' QoL. Mothers of children with CZS may experience heightened anxiety, depression, and overall reduced QoL, while many individuals with GBS struggle to regain the QoL they had pre-illness. The limited availability of high-quality data, primarily relying on case reports, highlights the need for more comprehensive research to establish clear causal links and better understand the long-term consequences of ZIKV-related neurological complications. Prioritizing research on long-term effects, management, and prevention strategies is crucial to improving patient outcomes and guiding future clinical care.
2026-07-07 | Arbovirus-Associated Guillain-Barré Syndrome: A Systematic Review and Meta-Analysis of Clinical Characteristics, Subtypes, and Vaccine Associations.
Arboviral infections are increasingly recognized as triggers of Guillain-Barré syndrome (GBS), yet the clinical spectrum, subtype distribution, and strength of association across different arboviruses remain incompletely characterized. This systematic review and meta-analysis evaluated the epidemiology, clinical features, and outcomes of arbovirus-associated GBS. PubMed, Scopus, Web of Science, and Google Scholar were searched through early 2025 following PRISMA 2020 guidelines. Observational studies were included in the quantitative meta-analysis, while case reports and case series were synthesized qualitatively. Random-effects models were used to estimate pooled prevalence, odds ratios, and clinical outcomes. One hundred studies were included, comprising 74 case reports/case series, 19 prevalence studies, and seven case-control studies. The pooled prevalence of GBS among individuals with arboviral infection was 1% (95% CI: 0.3%-3.3%), whereas 37% (95% CI: 22%-54%) of patients with GBS had laboratory evidence of recent arboviral infection. Arboviral co-infections occurred in 16% (95% CI: 8%-31%) of confirmed cases. Case-control studies demonstrated a significant association between Zika virus infection and GBS (OR = 8, 95% CI: 2-34). Qualitative synthesis showed frequent intensive care admission, mechanical ventilation, disability, and mortality. Demyelinating subtypes predominated in Zika virus-associated GBS, whereas axonal variants were more common following Japanese encephalitis virus infection. Multiple arboviruses are associated with GBS, with the strongest evidence for Zika virus. Arbovirus-associated GBS frequently results in severe neurological outcomes, highlighting the need for standardized diagnostics, enhanced surveillance, and prospective multicenter studies to improve understanding of disease mechanisms and optimize patient management.
Access all drug discovery articles and probability of success in trials forecasts:
Access all drug discovery articles and probability of success in trials forecasts:
Drug Discovery Landscape
11 orphan drug designations for Guillain-Barré syndrome.
11 orphan drug designations for Guillain-Barré syndrome.
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
Humanised IgG4 monoclonal antibody against C1q | antibodies | EMA | 2023-10-13 | — | Sinclair Regulatory Consulting Europe Limited |
Eculizumab | antibodies | EMA | 2022-10-11 | — | Alexion Europe SAS |
eculizumab | antibodies | FDA | 2022-09-28 | — | Alexion Pharmaceuticals, Inc. |
imlifidase | proteins | FDA | 2018-02-14 | — | Hansa Medical AB |
humanized IgG4 monoclonal antibody binding to human C1q | antibodies | FDA | 2017-01-19 | — | Annexon, Inc. |
Recombinant protein derived from the saliva of the Ornithodoros moubata tick | proteins | EMA | 2016-06-27 | — | Akari Malta Limited |
nomacopan | proteins | FDA | 2016-05-10 | — | Akari Therapeutics Plc |
immune globulin (human) | antibodies | FDA | 2010-03-02 | — | Octapharma USA, Inc. |
Fampridine | small molecules | EMA | 2007-07-10 | — | Ulrich Granzer |
4-Aminopyridine | small molecules | FDA | 2005-12-14 | — | Merz Pharmaceuticals LLC |
Immune Globulin Intravenous (human) | antibodies | FDA | 2004-05-04 | — | ZLB Bioplasma AG |
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