RARE DISEASE
Lyme disease
Lyme disease
Lyme disease
Synonyms: Lyme borreliosis
Synonyms: Lyme borreliosis
Synonyms: Lyme borreliosis
Drug discovery
0
drugs
With orphan designations
Overview
Lyme disease, caused by Borrelia burgdorferi and transmitted via blacklegged ticks, is the most common vector-borne illness in the U.S. Early localized infection often presents with erythema migrans rash, fever, and fatigue. Untreated cases may progress to disseminated arthritis, neurologic deficits, or cardiac complications. Diagnosis relies on clinical suspicion, exposure history, and serologic testing, while treatment focuses on prompt antibiotic therapy to prevent chronic sequelae [1][4][5][19].
Population
Bimodal age distribution (peaks at 5–14 and 45–64 years), with slight male predominance [1][6][18].
Higher incidence in White individuals, but racial/ethnic minorities face delayed diagnoses and more severe manifestations [2][12].
Endemic in Northeast, mid-Atlantic, and upper Midwest U.S. regions [5][10].
Therapies
First-line: Oral doxycycline (10–21 days) for early/localized disease; IV ceftriaxone for neurologic/cardiac involvement [3][7][11].
Post-treatment Lyme disease (PTLDS) management prioritizes symptom relief, not prolonged antibiotics [13][19][20].
Single-dose doxycycline prophylaxis recommended for high-risk tick bites in endemic areas [7][19].
Categories: rare infectious diseases, rare neurological diseases
Research Papers
2,194 drug discovery papers about Lyme disease, with 2 first-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
2,194 drug discovery papers about Lyme disease, with 2 first-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
2026-08-13 | Lyme Disease and Wellness Innovation
Lyme disease is a complex, multi-system illness caused primarily by Borrelia burgdorferi and transmitted through the bite of infected ticks.While early-stage Lyme disease may present as an acute infection with recognizable symptoms and is often responsive to timely antibiotic intervention, delayed diagnosis or incomplete treatment can allow the condition to progress into a chronic and debilitating state for a subset of individuals.In these cases, patients may experience persistent and relapsing symptoms that affect neurological, musculoskeletal, cardiovascular, gastrointestinal, and immune systems, significantly impairing quality of life.Conventional medical management of Lyme disease has historically focused on antimicrobial therapy as the primary line of treatment.However, a growing body of clinical and translational research suggests that ongoing symptoms in chronic or post-treatment Lyme disease are not solely attributable to active infection.Instead, they are increasingly understood to involve downstream biological disruptions, including immune system dysregulation, chronic low-grade inflammation, mitochondrial dysfunction, oxidative stress, autonomic imbalance, and alterations in the gut microbiome.These interrelated mechanisms can perpetuate fatigue, pain, cognitive dysfunction, mood disturbances, metabolic instability, and impaired detoxification capacity long after antimicrobial therapy has concluded.This white paper explores Lyme disease through an integrative biomedical and wellness-based lens, emphasizing the importance of addressing both pathogen-related factors and host resilience.It examines how targeted nutritional and botanical interventions specifically the strategic use of vitamins, minerals, antioxidants, and herbal compounds may help modulate inflammatory signaling, support mitochondrial energy production, enhance immune regulation, reduce oxidative burden, and restore gut and barrier integrity.When thoughtfully integrated alongside conventional medical care, these approaches may contribute to improved physiological adaptability, symptom management, and long-term recovery outcomes.By bridging conventional infectious disease management with systems-based nutritional and metabolic support, this paper aims to provide a more comprehensive framework for understanding and supporting individuals affected by Lyme disease.The goal is not to replace standard medical treatment, but to complement it with evidence-informed strategies that address the complex biological terrain underlying chronic symptoms, ultimately promoting greater resilience, functional recovery, and overall wellness.PriMera Scientific Surgical Research and Practice https://primerascientific.com/pssrp Lyme Disease and Wellness Innovation 47
2026-08-09 | Borrelial Lymphocytoma of the Ankle: An Unusual Presentation of Lyme Borreliosis.
Borrelial lymphocytoma is an uncommon cutaneous manifestation of Lyme borreliosis and is considered a localized reactive B-cell pseudolymphoma induced by infection with members of the Borrelia burgdorferi sensu lato complex. The condition most commonly affects the ear lobe, nipple-areolar region, and scrotum, whereas involvement of the ankle is rarely reported. We report the case of a 47-year-old man who presented with a persistent erythematous papule adjacent to the lateral malleolus following a tick bite sustained approximately one year earlier. The lesion persisted despite several topical treatments. The patient did not recall a preceding episode of erythema migrans and had no extracutaneous manifestations suggestive of Lyme borreliosis. Borrelia serology performed by enzyme-linked immunosorbent assay (ELISA) revealed positive Borrelia IgG antibodies, which were subsequently confirmed by immunoblot. Histopathological examination of the skin biopsy demonstrated a dense dermal lymphoid infiltrate with reactive lymphoid follicles and numerous plasma cells, findings consistent with borrelial lymphocytoma in the appropriate clinicopathological context. Treatment with doxycycline (100 mg twice daily for three weeks) resulted in marked clinical improvement and near-complete resolution of the lesion within a few weeks. No recurrence was observed during six months of follow-up. This case highlights the rare occurrence of borrelial lymphocytoma involving the lateral malleolus, an unusual anatomical location that may delay diagnosis because of its atypical presentation. It also emphasizes the importance of clinicopathological correlation, integrating the clinical history, serological findings, and histopathological features, in establishing an accurate diagnosis and distinguishing borrelial lymphocytoma from other cutaneous pseudolymphomatous and lymphoproliferative disorders.
2026-07-31 | Neuroborreliosis complicated by severe bilateral sensorineural hearing loss.
Neuroborreliosis (NB) is a neurologic complication of Lyme disease that can cause sudden hearing loss, which can in some cases be bilateral. A 61-year-old woman consulted for bilateral sudden hearing loss with headache and earache, following an episode of fever. Interview found a skin lesion suggestive of migratory erythema, which had developed 3months previously after a walk in a German forest and had not initially been treated. Examination revealed severe sensorineural hearing loss and lymphocytic meningitis. Blood tests found long-standing IgM and IgG positive Borrelia burgdorferi infection with intrathecal synthesis of IgM and IgG antibodies. Corticosteroid treatment and ceftriaxone was initiated, and normalized hearing within 3weeks. Hearing impairment in NB may involve inflammatory, immunologic or cytotoxic mechanisms affecting hair cells. Early treatment with corticosteroids and targeted antibiotics should be prescribed when NB is suspected, to optimize the chances of recovery. In endemic geographic areas, signs of Lyme disease should be screened for in case of sudden hearing loss.
2026-07-30 | Gustatory function of Aedes aegypti opsins and TRPA1 in avoiding a natural insect repellent.
The mosquito, Aedes aegypti, spreads viruses that cause dengue, yellow fever, and other devastating diseases. To identify candidate repellents that suppress blood feeding by Aedes aegypti, we conducted an in vitro screen of natural chemicals for activators of two visual opsins, Op1 and Op2, which we found are expressed at very high levels in gustatory organs. We found that Op1 and Op2 are chemical sensors since they are activated by flavonoids, including catechin. We devised an artificial membrane that mimics multiple properties of skin (VectaDerm), which we used on blood feeders, leading to the demonstration that catechin suppresses blood feeding. This behavior was disrupted in op1,op2 double mutants and in trpA1 mutants. High catechin concentrations directly activated TRPA1 and suppressed blood feeding independent of the opsins, while suppression by lower catechin levels depended on the opsins and TRPA1. Roles for opsins and TRPA1 in avoiding catechin are conserved in Drosophila. TRPA1 expressed in the tick vector for Lyme disease, Ixodes scapularis, is also activated by catechin, and I. scapularis is repelled by catechin. Since catechin is safe to humans, we propose that it could be added to repellent formulations to more effectively suppress biting by mosquitoes.
2026-07-28 | Prevalence and Associated Factors of Borrelia burgdorferi Sensu Lato Exposure in Humans and Infection in Questing Ixodes Ticks in China: A Systematic Review and Meta-Analysis.
Lyme borreliosis has been reported across China, but data on Borrelia burgdorferi sensu lato exposure in humans and infection in questing Ixodes ticks remains fragmented and heterogeneous. This systematic review and meta-analysis aimed to estimate the pooled seroprevalence of B. burgdorferi sensu lato exposure in humans and infection prevalence in questing or unfed Ixodes ticks in China and to explore potential sources of heterogeneity. Six English- and Chinese-language databases were searched from inception to 10 September 2025. Eligible cross-sectional studies reported both the number examined and the number positive, with a minimum sample size of 30. Human analyses were restricted to serological evidence, whereas tick analyses included questing or unfed Ixodes ticks collected from vegetation or the environment. Random-effects models, subgroup analyses, and univariate random-effects meta-regression were applied. A total of 186 studies were included, of which 167 reported human data, 22 reported PCR-based data from questing Ixodes ticks, and 3 reported both human and tick data. The pooled human seroprevalence was 7% (95% CI: 6-8%), and the pooled PCR-confirmed infection prevalence in questing Ixodes ticks was 20% (95% CI: 10-32%). Substantial heterogeneity was observed in both analyses. For questing Ixodes ticks, infection prevalence showed marked geographical heterogeneity, with the highest pooled estimate observed in Northeastern China. In univariate meta-regression, Northern China showed a significantly lower estimate than Northeastern China, whereas sampling year, reported Borrelia genospecies, and Ixodes species were not significantly associated with pooled prevalence. Leave-one-out analyses indicated that the pooled estimates were not driven by any single study. These findings demonstrate that human exposure and environmental circulation of B. burgdorferi sensu lato have been documented across multiple regions of China but remain highly heterogeneous.
2026-08-13 | Lyme Disease and Wellness Innovation
Lyme disease is a complex, multi-system illness caused primarily by Borrelia burgdorferi and transmitted through the bite of infected ticks.While early-stage Lyme disease may present as an acute infection with recognizable symptoms and is often responsive to timely antibiotic intervention, delayed diagnosis or incomplete treatment can allow the condition to progress into a chronic and debilitating state for a subset of individuals.In these cases, patients may experience persistent and relapsing symptoms that affect neurological, musculoskeletal, cardiovascular, gastrointestinal, and immune systems, significantly impairing quality of life.Conventional medical management of Lyme disease has historically focused on antimicrobial therapy as the primary line of treatment.However, a growing body of clinical and translational research suggests that ongoing symptoms in chronic or post-treatment Lyme disease are not solely attributable to active infection.Instead, they are increasingly understood to involve downstream biological disruptions, including immune system dysregulation, chronic low-grade inflammation, mitochondrial dysfunction, oxidative stress, autonomic imbalance, and alterations in the gut microbiome.These interrelated mechanisms can perpetuate fatigue, pain, cognitive dysfunction, mood disturbances, metabolic instability, and impaired detoxification capacity long after antimicrobial therapy has concluded.This white paper explores Lyme disease through an integrative biomedical and wellness-based lens, emphasizing the importance of addressing both pathogen-related factors and host resilience.It examines how targeted nutritional and botanical interventions specifically the strategic use of vitamins, minerals, antioxidants, and herbal compounds may help modulate inflammatory signaling, support mitochondrial energy production, enhance immune regulation, reduce oxidative burden, and restore gut and barrier integrity.When thoughtfully integrated alongside conventional medical care, these approaches may contribute to improved physiological adaptability, symptom management, and long-term recovery outcomes.By bridging conventional infectious disease management with systems-based nutritional and metabolic support, this paper aims to provide a more comprehensive framework for understanding and supporting individuals affected by Lyme disease.The goal is not to replace standard medical treatment, but to complement it with evidence-informed strategies that address the complex biological terrain underlying chronic symptoms, ultimately promoting greater resilience, functional recovery, and overall wellness.PriMera Scientific Surgical Research and Practice https://primerascientific.com/pssrp Lyme Disease and Wellness Innovation 47
2026-08-09 | Borrelial Lymphocytoma of the Ankle: An Unusual Presentation of Lyme Borreliosis.
Borrelial lymphocytoma is an uncommon cutaneous manifestation of Lyme borreliosis and is considered a localized reactive B-cell pseudolymphoma induced by infection with members of the Borrelia burgdorferi sensu lato complex. The condition most commonly affects the ear lobe, nipple-areolar region, and scrotum, whereas involvement of the ankle is rarely reported. We report the case of a 47-year-old man who presented with a persistent erythematous papule adjacent to the lateral malleolus following a tick bite sustained approximately one year earlier. The lesion persisted despite several topical treatments. The patient did not recall a preceding episode of erythema migrans and had no extracutaneous manifestations suggestive of Lyme borreliosis. Borrelia serology performed by enzyme-linked immunosorbent assay (ELISA) revealed positive Borrelia IgG antibodies, which were subsequently confirmed by immunoblot. Histopathological examination of the skin biopsy demonstrated a dense dermal lymphoid infiltrate with reactive lymphoid follicles and numerous plasma cells, findings consistent with borrelial lymphocytoma in the appropriate clinicopathological context. Treatment with doxycycline (100 mg twice daily for three weeks) resulted in marked clinical improvement and near-complete resolution of the lesion within a few weeks. No recurrence was observed during six months of follow-up. This case highlights the rare occurrence of borrelial lymphocytoma involving the lateral malleolus, an unusual anatomical location that may delay diagnosis because of its atypical presentation. It also emphasizes the importance of clinicopathological correlation, integrating the clinical history, serological findings, and histopathological features, in establishing an accurate diagnosis and distinguishing borrelial lymphocytoma from other cutaneous pseudolymphomatous and lymphoproliferative disorders.
2026-07-31 | Neuroborreliosis complicated by severe bilateral sensorineural hearing loss.
Neuroborreliosis (NB) is a neurologic complication of Lyme disease that can cause sudden hearing loss, which can in some cases be bilateral. A 61-year-old woman consulted for bilateral sudden hearing loss with headache and earache, following an episode of fever. Interview found a skin lesion suggestive of migratory erythema, which had developed 3months previously after a walk in a German forest and had not initially been treated. Examination revealed severe sensorineural hearing loss and lymphocytic meningitis. Blood tests found long-standing IgM and IgG positive Borrelia burgdorferi infection with intrathecal synthesis of IgM and IgG antibodies. Corticosteroid treatment and ceftriaxone was initiated, and normalized hearing within 3weeks. Hearing impairment in NB may involve inflammatory, immunologic or cytotoxic mechanisms affecting hair cells. Early treatment with corticosteroids and targeted antibiotics should be prescribed when NB is suspected, to optimize the chances of recovery. In endemic geographic areas, signs of Lyme disease should be screened for in case of sudden hearing loss.
2026-07-30 | Gustatory function of Aedes aegypti opsins and TRPA1 in avoiding a natural insect repellent.
The mosquito, Aedes aegypti, spreads viruses that cause dengue, yellow fever, and other devastating diseases. To identify candidate repellents that suppress blood feeding by Aedes aegypti, we conducted an in vitro screen of natural chemicals for activators of two visual opsins, Op1 and Op2, which we found are expressed at very high levels in gustatory organs. We found that Op1 and Op2 are chemical sensors since they are activated by flavonoids, including catechin. We devised an artificial membrane that mimics multiple properties of skin (VectaDerm), which we used on blood feeders, leading to the demonstration that catechin suppresses blood feeding. This behavior was disrupted in op1,op2 double mutants and in trpA1 mutants. High catechin concentrations directly activated TRPA1 and suppressed blood feeding independent of the opsins, while suppression by lower catechin levels depended on the opsins and TRPA1. Roles for opsins and TRPA1 in avoiding catechin are conserved in Drosophila. TRPA1 expressed in the tick vector for Lyme disease, Ixodes scapularis, is also activated by catechin, and I. scapularis is repelled by catechin. Since catechin is safe to humans, we propose that it could be added to repellent formulations to more effectively suppress biting by mosquitoes.
2026-07-28 | Prevalence and Associated Factors of Borrelia burgdorferi Sensu Lato Exposure in Humans and Infection in Questing Ixodes Ticks in China: A Systematic Review and Meta-Analysis.
Lyme borreliosis has been reported across China, but data on Borrelia burgdorferi sensu lato exposure in humans and infection in questing Ixodes ticks remains fragmented and heterogeneous. This systematic review and meta-analysis aimed to estimate the pooled seroprevalence of B. burgdorferi sensu lato exposure in humans and infection prevalence in questing or unfed Ixodes ticks in China and to explore potential sources of heterogeneity. Six English- and Chinese-language databases were searched from inception to 10 September 2025. Eligible cross-sectional studies reported both the number examined and the number positive, with a minimum sample size of 30. Human analyses were restricted to serological evidence, whereas tick analyses included questing or unfed Ixodes ticks collected from vegetation or the environment. Random-effects models, subgroup analyses, and univariate random-effects meta-regression were applied. A total of 186 studies were included, of which 167 reported human data, 22 reported PCR-based data from questing Ixodes ticks, and 3 reported both human and tick data. The pooled human seroprevalence was 7% (95% CI: 6-8%), and the pooled PCR-confirmed infection prevalence in questing Ixodes ticks was 20% (95% CI: 10-32%). Substantial heterogeneity was observed in both analyses. For questing Ixodes ticks, infection prevalence showed marked geographical heterogeneity, with the highest pooled estimate observed in Northeastern China. In univariate meta-regression, Northern China showed a significantly lower estimate than Northeastern China, whereas sampling year, reported Borrelia genospecies, and Ixodes species were not significantly associated with pooled prevalence. Leave-one-out analyses indicated that the pooled estimates were not driven by any single study. These findings demonstrate that human exposure and environmental circulation of B. burgdorferi sensu lato have been documented across multiple regions of China but remain highly heterogeneous.
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