Medical therapy in spinal muscular atrophy: a realistic expectation?
The hereditary spinal muscular atrophies (SMA) type I-III belong to those diseases for which even the thought of medical therapy seems forbidden. Two neurotrophic factors are, however, now known to exert a markedly stimulating effect on survival of motor neurons in vivo! In principle such factors may become available by recombinant DNA techniques for experiments in animal models of SMA and if these experiments are successful for clinical trials in man. Medical therapy in SMA should aim primarily at patients early in the rapidly progressive phase of their disease, before massive loss of motoneuron has taken place.
Jennekens
F G
FG
Department of Neurology, University Hospital, University of Utrecht, The Netherlands.
D000324:Adrenocorticotropic Hormone / Q000627:therapeutic use*; D000328:Adult; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D009046:Motor Neurons / Q000187:drug effects; D009134:Muscular Atrophy, Spinal / Q000188:drug therapy*; D009410:Nerve Degeneration / Q000187:drug effects*; D009414:Nerve Growth Factors / Q000627:therapeutic use*; D010446:Peptide Fragments / Q000627:therapeutic use*; D014897:Spinal Muscular Atrophies of Childhood / Q000188:drug therapy; D000521:alpha-MSH / Q000627:therapeutic use*
D009414:Nerve Growth Factors; D010446:Peptide Fragments; C028024:ACTH (4-9); D000521:alpha-MSH; D000324:Adrenocorticotropic Hormone