title: [Recent clinical knowledge of cytoplasmic substances in myogenic and neuromuscular diseases, especially in muscular dystrophy, Duchenne type].
abstract: Up to now there has been no known causal therapy of numerous myogenic and neuromuscular diseases. Among other disorders the genetic muscular dystrophies--especially the malignant Duchenne type, as well as the spinal and neuromuscular dystrophies--cause considerable problems in therapy. Nevertheless there are possibilities of positively influencing the clinical picture and course of the disease by skillful physical training and symptomatic medication. It is sometimes possible, by means of such therapy, to obtain--within limitations imposed by irreparable lesions-stabilization of the clinical state with prolongation of walking ability and amelioration of other motor functions. Nevertheless, such improvement is only transient, because in all patients the process progresses sooner or later. Significant effects were registered of so-called cytoplasmic substances or "biological response modifiers" (particles of protoplasm consisting of nuclei, mitochondria, microsomes and membranes of fresh organic material from healthy young animals and animal fetuses) in an uncommonly large collective of boys with Duchenne muscular dystrophy, as evaluated by follow-up control of the serum enzyme index and creatinine creatine excretion rate. The role of calcium and the possible effect of proteinase-inhibitors are discussed among other pathogenetic aspects. Reference is also made to potential therapeutic effects of cytoplasmic substances in spinal and neuromuscular atrophies on the basis of initial preliminary clinical experience in this field.
mesh_terms: D003593:Cytoplasm / Q000032:analysis*; D004734:Energy Metabolism; D004798:Enzymes / Q000097:blood; D006801:Humans; D009135:Muscular Diseases / Q000097:blood* / Q000473:pathology; D009136:Muscular Dystrophies / Q000097:blood* / Q000473:pathology; D009468:Neuromuscular Diseases / Q000097:blood* / Q000473:pathology
chemical_list: D004798:Enzymes
vernacular_title: Neuere klinische Erkenntnisse mit zytoplasmatischen Substanzen bei myogenen und neuromuskulären Erkrankungen, insbesondere bei der Muskeldystrophie, Typ Duchenne.