Gene/Protein Disease Symptom Drug Enzyme Compound
Pivot Concepts:   Target Concepts:
Query: UMLS:C0026838 (spasticity)
 6,471 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

Neruomyelitis optica (NMO) is an inflammatory disease of the central nervous system characterized by severe optic neuritis and transverse myelitis, usually with a relapsing course. It is crucial that treatment is started as early as possible to avoid new relapses and further disability. Treatment of NMO spectrum disorders is divided into two objectives: one is to control the inflammatory damage in acute attacks and the other one is a maintenance treatment to avoid relapses. The former is based on high-dose intravenous corticosteroids and plasmapheresis, the latter is based on low-dose corticosteroids and immunosuppressants. High-dose intravenous methylprednisolone is the first-line therapy for acute exacerbations of NMO spectrum disorders. Plasmapheresis should be started soon if corticosteroid is not efficacious. Maintenance therapy is based on low-dose oral corticosteroids and non-specific immunosuppressant drugs, like azathioprine, tacrolimus, mycophenolate mofetil and mitoxantrone. New therapy strategies using monoclonal antibodies like rituximab; an anti-CD20 monoclonal antibody, and eculizumab; an anti-C5 monoclonal antibody, can also prevent relapse in NMO. On the other hand, interferon-beta, natalizumab and fingolimod, a first-line disease modifying drug of multiple sclerosis, is not effective in NMO spectrum disorders. Treatment of the symptom for spasticity, pain, dysuria may significantly improve the quality of life of the NMO patient.
 ...
PMID:[Treatment of neuromyelitis optica]. 2377 90

Krabbe disease, or globoid cell leukodystrophy, is a rare disorder caused by deficient galactosylceramidase activity and loss of myelin-forming oligodendrocytes, resulting in progressive demyelination and severely impaired motor function. Disease symptoms in humans appear within 3-6 months of age (early infantile) and manifest as marked irritability, spasticity, and seizures. The disease is often fatal by the second year of life, with few effective treatment options. Herein we evaluated the therapeutic potential of mesenchymal stem cells (MSCs) administered intracranially to a 1-month-old rhesus macaque diagnosed with severe early-onset Krabbe disease that displayed neurologic and behavioral symptoms similar to those of human patients. The infant was subjected to physical and neurological behavior examinations and nerve conduction velocity tests to assess efficacy, and outcomes were compared with age-matched normal infants and Krabbe-affected rhesus monkeys with late-onset disease. Changes in major blood lymphocyte populations were also monitored to assess host immune cell responses. MSC administration resulted in transient improvements in coordination, ambulation, cognition, and large motor skills, which correlated with increased peripheral nerve conduction velocities and decreased latencies. Improvements also corresponded to transient increases in peripheral blood lymphocyte counts, but secondary challenge failed to elicit allo-antibody production. Nevertheless, white cell and neutrophil counts showed dramatic increases, and CD20+ B cell counts underwent a precipitous decline at late stages of disease progression. Correlative data linking MSC administration to transient improvements in motor function suggest that MSCs should be evaluated further as an experimental therapy for rare neurodegenerative diseases. Stem Cells Translational Medicine 2017;6:99-109.
 ...
PMID:Mesenchymal Stem Cells Yield Transient Improvements in Motor Function in an Infant Rhesus Macaque with Severe Early-Onset Krabbe Disease. 2817 Jan 89