Gene/Protein Disease Symptom Drug Enzyme Compound
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Query: UMLS:C0002736 (amyotrophic lateral sclerosis)
 19,048 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

Diffusion characteristics of the pyramidal tract were assessed in nine patients who had clinical evidence of pyramidal tract dysfunction, utilizing lambda chart analysis (LCA). The underlying pathologic process of tract dysfunction was varied and included Pelizaeus-Merzbacher disease (PMD), Alexander disease, adrenoleukodystrophy (adrenomyeloneuropathy (AMD) type and cerebral type), amyotrophic lateral sclerosis (ALS), and Wallerian degeneration (WD). While pyramidal tract diffusion characteristics in WD indicated a pathological process characterized by replacement of normal fibers by smaller cellular component such as degenerated small fibers and/or gliosis, pyramidal tract diffusion characteristics in patients with PMD, Alexander disease, and adreno leukodystrophy of the cerebral type indicated a pathological process characterized by replacement of normal fibers by larger cellular components such as spheroids or edematous space. Pyramidal tract diffusion characteristics of patients with ALS or adrenoleukodystrophy of AMD type were relatively intact suggesting a pathological process characterized by relatively preserved structural architecture. These findings are highly consistent with known pathophysiological indices and indicate the feasibility of the clinical utility of LCA for assessing pyramidal tract physiology.
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PMID:Feasibility study of single region lambda chart analysis for pyramidal tract physiology. 1458 99

Currently, there are five phase I clinical trials of recombinant adeno-associated viral vectors for the treatment of neurological disorders that are approved or likely to be approved shortly. Two trials are testing different strategies to treat Parkinson's disease (PD), the third trial is aimed at treating Canavan's disease, a pediatric leukodystrophy, the fourth trial targets Alzheimer's disease (AD), and the fifth will attempt to target the lysosomal storage disorder, Batten's disease. All four clinical trials rely on the de novo expression of an enzyme or a trophic factor to correct neuropathology. Ironically, the theories used to choose enzymes for the two PD trials were widely divergent, whereas the enzymatic strategy used for one of the PD trials and the Canavan's trial have remarkable similarities. Other gene therapy treatment strategies for PD and other disorders, such as amyotrophic lateral sclerosis, are also on the horizon.
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PMID:Clinical trials in neurological disorders using AAV vectors: promises and challenges. 1553 49