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The effect of oral betaine on vertebral body bone density in pyridoxine-non-responsive homocystinuria

W A Gahl, I Bernardini, S Chen, D Kurtz, K Horvath
Journal of inherited metabolic disease 1988 11(3):291-298

Bibliography

PubMed
PMID 3148071
Funding
Funding was not specified in the indexed record or accessible funding metadata.
Competing interests
A competing-interest declaration was not available in the indexed record or accessible disclosure metadata.

Study snapshot

DesignDouble-blind controlled crossover study
ModelHuman participants
Sample5 patients with pyridoxine-nonresponsive homocystinuria
InterventionBetaine 3 g twice daily versus placebo
Duration2-year crossover protocol
EndpointsHomocystine; Vertebral bone density

What the study showed, in plain terms

Published in 1988, this source examined prescription betaine and inherited metabolic disorders. Design: Double-blind controlled crossover study. Sample/model: 5 patients with pyridoxine-nonresponsive homocystinuria. The exposure or intervention was Betaine 3 g twice daily versus placebo. Follow-up or treatment period: 2-year crossover protocol.

Betaine lowered homocystine, but vertebral bone density did not significantly improve.

Extremely small rare-disease study; biochemical benefit did not establish a bone-density effect.

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Key findings

  • Betaine lowered homocystine, but vertebral bone density did not significantly improve.
  • The recorded outcome domains were Homocystine; Vertebral bone density.
  • The result applies to the tested exposure: Betaine 3 g twice daily versus placebo; 2-year crossover protocol.

What this study can and cannot tell us

  • Extremely small rare-disease study; biochemical benefit did not establish a bone-density effect.
  • An individual controlled trial must be interpreted alongside its comparator, sample size, endpoint selection and independent replication. It does not validate a different commercial formulation or a general longevity claim.
  • Source basis: Indexed abstract and bibliographic data. Missing method details, funding information or participant denominators are not inferred.

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