RARE DISEASE
Acute transverse myelitis
Acute transverse myelitis
Acute transverse myelitis
Drug discovery
2
drugs
With orphan designations
Overview
Acute Transverse Myelitis (ATM) is an acute inflammatory disorder affecting spinal cord gray and white matter, typically presenting with bilateral motor, sensory, and autonomic deficits below the lesion. Common etiologies include multiple sclerosis, neuromyelitis optica spectrum disorder (NMOSD), infections, autoimmune conditions, or idiopathic causes. Diagnosis relies on MRI, cerebrospinal fluid analysis, and exclusion of compressive lesions. Initial treatment involves high-dose IV corticosteroids and plasma exchange for refractory cases. Prognosis varies, with one-third recovering fully, one-third retaining residual deficits, and one-third experiencing severe disability [1][2][3][5][7].
Burden
Disability: ~33% require ambulatory aids or lifelong care; severe bladder/bowel dysfunction in 80–94% [1][3][5].
Mortality: Up to 10% in longitudinal studies, often due to comorbidities or complications [4][7].
Economic impact: High costs from prolonged rehabilitation, missed work, and chronic care needs [4][7].
Therapies
Categories: rare neurological diseases
Research Papers
395 drug discovery papers about Acute transverse myelitis, with 2 first-in-class and 3 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
395 drug discovery papers about Acute transverse myelitis, with 2 first-in-class and 3 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
2026-06-26 | Hantavirus infection: Neurologic manifestations should not be overlooked.
Hantavirus infection is primarily associated with hemorrhagic fever with renal syndrome (HFRS) and hantavirus cardiopulmonary syndrome (HCPS), with predominant renal and pulmonary involvement. However, neurological manifestations affecting both the central nervous system (CNS) and peripheral nervous system (PNS) are increasingly recognized. We conducted a narrative review of the literature to summarize the current evidence regarding hantavirus-associated neurological involvement. Reported CNS manifestations included encephalitis, encephalopathy, seizures, meningitis, neurocognitive alterations, posterior reversible encephalopathy syndrome, transverse myelitis, and cerebral hemorrhage. PNS involvement appeared less frequent and included Guillain-Barré syndrome, cranial nerve palsies, neuropathic pain, and sensory disturbances. Neuroimaging findings were heterogeneous, while cerebrospinal fluid analysis often demonstrated nonspecific inflammatory changes. Advanced molecular techniques such as metagenomic next-generation sequencing may improve diagnostic sensitivity, particularly in immunocompromised patients. Current evidence suggests that neurological involvement may result from endothelial dysfunction, neuroinflammation, immune-mediated injury, blood-brain barrier disruption, and, in selected cases, direct viral neuroinvasion. Greater clinical awareness is needed to improve recognition of neurological complications during hantavirus infection. Further prospective studies are required to better define the epidemiology, pathogenesis, and optimal diagnostic approaches of hantavirus-associated neurological disease.
2026-06-02 | Spinal venous hypertension secondary to nutcracker syndrome-a previously unreported cause of congestive myelopathy.
We report a case of myelopathy associated with nutcracker syndrome. Clinical and imaging findings indicate that nutcracker syndrome caused left renal vein stenosis, resulting in spinal venous hypertension. A 15-year-old girl presented with abnormal sensations in both lower limbs, followed by progressive motor impairment and bowel and bladder dysfunction. Magnetic resonance imaging (MRI) revealed an abnormal signal in the spinal cord at the T12-L1 level. Although she was initially diagnosed with acute transverse myelitis (ATM) and treated with intravenous steroids, cerebrospinal fluid (CSF) analysis showed no signs of inflammation. Further evaluation with Doppler ultrasound and left renal arteriovenous angiography suggested that nutcracker syndrome led to compression of the left renal vein, with aberrant venous branches draining into the spinal venous system. This resulted in spinal venous hypertension and subsequent spinal cord injury. Initial treatment with corticosteroids and immunoglobulins was discontinued due to disease progression. After identifying a left renal vein vascular malformation at an external hospital, balloon dilation of the left renal vein was performed, yielding mild symptomatic relief. Subsequent interventional embolization of the malformed vessels temporarily improved symptoms, though her condition later deteriorated. Ultimately, she underwent laparoscopic robot-assisted external stent placement in the left renal vein at our institution. During the three-month postoperative follow-up, sensory function gradually improved and the clinical condition stabilized. To our knowledge, this is the first reported case of spinal venous hypertension syndrome secondary to nutcracker syndrome, presenting as myelopathy. We emphasize that in cases of acute myelitis or spinal cord disease of unknown etiology, screening for extramedullary vascular abnormalities-such as left renal artery or vein compression or malformations-should be considered, particularly when inflammatory markers are absent or treatment response is atypical.
2026-06-01 | Spinal H-Sign in Syphilitic Myelitis Mimicking MOGAD.
Longitudinally extensive transverse myelitis (LETM) is classically associated with aquaporin-4 antibody-mediated neuromyelitis optica spectrum disorder and myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD). The "H-sign," reflecting gray matter involvement, is considered suggestive of MOGAD. However, infectious etiologies, including neurosyphilis, may rarely mimic autoimmune inflammatory myelopathies radiologically. A 50-year-old man presented with 6 months of erectile dysfunction and urinary retention, followed by progressive gait instability. Examination revealed spastic paraparesis with tetrapyramidal signs, saddle hypoesthesia, and impaired vibration sense. Thoracic spine MRI demonstrated LETM from T4 to T10 with central gray matter involvement forming the "H-sign." Postcontrast sequences showed intense pial and intramedullary enhancement with inversion of signal intensity ("flip-flop sign"), suggestive of inflammatory meningomyelitis. Brain MRI was normal. Serum treponemal tests were reactive, and cerebrospinal fluid analysis revealed lymphocytic pleocytosis, elevated protein, and positive VDRL, confirming neurosyphilis. MOG-IgG and AQP4-IgG were negative using validated cell-based assays. The patient received intravenous benzylpenicillin combined with high-dose corticosteroids, resulting in significant clinical improvement and partial radiologic resolution at 6 months. Neurosyphilis is a rare but treatable cause of LETM and may radiologically mimic antibody-mediated demyelinating disorders, including MOGAD. The coexistence of the "H-sign" and the flip-flop enhancement pattern highlights the diagnostic overlap between infectious and autoimmune myelitis. Careful exclusion of infectious etiologies remains essential in patients presenting with LETM to ensure appropriate therapy and avoid unnecessary immunosuppression.
2026-06-26 | Hantavirus infection: Neurologic manifestations should not be overlooked.
Hantavirus infection is primarily associated with hemorrhagic fever with renal syndrome (HFRS) and hantavirus cardiopulmonary syndrome (HCPS), with predominant renal and pulmonary involvement. However, neurological manifestations affecting both the central nervous system (CNS) and peripheral nervous system (PNS) are increasingly recognized. We conducted a narrative review of the literature to summarize the current evidence regarding hantavirus-associated neurological involvement. Reported CNS manifestations included encephalitis, encephalopathy, seizures, meningitis, neurocognitive alterations, posterior reversible encephalopathy syndrome, transverse myelitis, and cerebral hemorrhage. PNS involvement appeared less frequent and included Guillain-Barré syndrome, cranial nerve palsies, neuropathic pain, and sensory disturbances. Neuroimaging findings were heterogeneous, while cerebrospinal fluid analysis often demonstrated nonspecific inflammatory changes. Advanced molecular techniques such as metagenomic next-generation sequencing may improve diagnostic sensitivity, particularly in immunocompromised patients. Current evidence suggests that neurological involvement may result from endothelial dysfunction, neuroinflammation, immune-mediated injury, blood-brain barrier disruption, and, in selected cases, direct viral neuroinvasion. Greater clinical awareness is needed to improve recognition of neurological complications during hantavirus infection. Further prospective studies are required to better define the epidemiology, pathogenesis, and optimal diagnostic approaches of hantavirus-associated neurological disease.
2026-06-02 | Spinal venous hypertension secondary to nutcracker syndrome-a previously unreported cause of congestive myelopathy.
We report a case of myelopathy associated with nutcracker syndrome. Clinical and imaging findings indicate that nutcracker syndrome caused left renal vein stenosis, resulting in spinal venous hypertension. A 15-year-old girl presented with abnormal sensations in both lower limbs, followed by progressive motor impairment and bowel and bladder dysfunction. Magnetic resonance imaging (MRI) revealed an abnormal signal in the spinal cord at the T12-L1 level. Although she was initially diagnosed with acute transverse myelitis (ATM) and treated with intravenous steroids, cerebrospinal fluid (CSF) analysis showed no signs of inflammation. Further evaluation with Doppler ultrasound and left renal arteriovenous angiography suggested that nutcracker syndrome led to compression of the left renal vein, with aberrant venous branches draining into the spinal venous system. This resulted in spinal venous hypertension and subsequent spinal cord injury. Initial treatment with corticosteroids and immunoglobulins was discontinued due to disease progression. After identifying a left renal vein vascular malformation at an external hospital, balloon dilation of the left renal vein was performed, yielding mild symptomatic relief. Subsequent interventional embolization of the malformed vessels temporarily improved symptoms, though her condition later deteriorated. Ultimately, she underwent laparoscopic robot-assisted external stent placement in the left renal vein at our institution. During the three-month postoperative follow-up, sensory function gradually improved and the clinical condition stabilized. To our knowledge, this is the first reported case of spinal venous hypertension syndrome secondary to nutcracker syndrome, presenting as myelopathy. We emphasize that in cases of acute myelitis or spinal cord disease of unknown etiology, screening for extramedullary vascular abnormalities-such as left renal artery or vein compression or malformations-should be considered, particularly when inflammatory markers are absent or treatment response is atypical.
2026-06-01 | Spinal H-Sign in Syphilitic Myelitis Mimicking MOGAD.
Longitudinally extensive transverse myelitis (LETM) is classically associated with aquaporin-4 antibody-mediated neuromyelitis optica spectrum disorder and myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD). The "H-sign," reflecting gray matter involvement, is considered suggestive of MOGAD. However, infectious etiologies, including neurosyphilis, may rarely mimic autoimmune inflammatory myelopathies radiologically. A 50-year-old man presented with 6 months of erectile dysfunction and urinary retention, followed by progressive gait instability. Examination revealed spastic paraparesis with tetrapyramidal signs, saddle hypoesthesia, and impaired vibration sense. Thoracic spine MRI demonstrated LETM from T4 to T10 with central gray matter involvement forming the "H-sign." Postcontrast sequences showed intense pial and intramedullary enhancement with inversion of signal intensity ("flip-flop sign"), suggestive of inflammatory meningomyelitis. Brain MRI was normal. Serum treponemal tests were reactive, and cerebrospinal fluid analysis revealed lymphocytic pleocytosis, elevated protein, and positive VDRL, confirming neurosyphilis. MOG-IgG and AQP4-IgG were negative using validated cell-based assays. The patient received intravenous benzylpenicillin combined with high-dose corticosteroids, resulting in significant clinical improvement and partial radiologic resolution at 6 months. Neurosyphilis is a rare but treatable cause of LETM and may radiologically mimic antibody-mediated demyelinating disorders, including MOGAD. The coexistence of the "H-sign" and the flip-flop enhancement pattern highlights the diagnostic overlap between infectious and autoimmune myelitis. Careful exclusion of infectious etiologies remains essential in patients presenting with LETM to ensure appropriate therapy and avoid unnecessary immunosuppression.
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Drug Discovery Landscape
2 orphan drug designations for Acute transverse myelitis.
2 orphan drug designations for Acute transverse myelitis.
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
dalfampridine | small molecules | FDA | 2019-11-25 | — | Magnum Therapeutics |
human glial restricted progenitor cells and their progeny | cell therapies | FDA | 2018-04-11 | — | Q Therapeutics, Inc. |
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