Tier 2 — strong

A study of the treatment of Rett syndrome with folate and betaine

Daniel G Glaze, Alan K Percy, Kathleen J Motil, Jane B Lane, Janet S Isaacs, Rebecca J Schultz, Judy O Barrish, Jeffrey L Neul, William E O'Brien, E O'Brian Smith
Journal of child neurology 2009 24(5):551-556

Bibliography

PubMed
PMID 19225139
PubMed Central
PMC2760386
DOI
10.1177/0883073808327827
Funding
Indexed grant support: K01 RR000188 — NCRR NIH HHS — United States; P30 HD038985 — NICHD NIH HHS — United States; M01 RR000188 — NCRR NIH HHS — United States; RR00188 — NCRR NIH HHS — United States; U54 HD061222 — NICHD NIH HHS — United States; M01 RR000032 — NCRR NIH HHS — United States; HD38985 — NICHD NIH HHS — United States; RR00032 — NCRR NIH HHS — United States; P01 HD040301 — NICHD NIH HHS — United States; HD40301 — NICHD NIH HHS — United States. The indexed list may not contain the complete funding declaration.
Competing interests
Reported disclosure: The authors have no conflicts of interest to disclose with regard to this article.

Study snapshot

DesignRandomized controlled trial
ModelHuman participants
Sample73 girls and women with MECP2-positive Rett syndrome; 68 completed
InterventionFolate plus betaine versus placebo
Duration12 months
EndpointsBreathing; Motor and behavioral function; Growth

What the study showed, in plain terms

Published in 2009, this source examined brain health and combination interventions. Design: Randomized controlled trial. Sample/model: 73 girls and women with MECP2-positive Rett syndrome; 68 completed. The exposure or intervention was Folate plus betaine versus placebo. Follow-up or treatment period: 12 months.

Objective measures did not show improvement. Parents reported subjective improvement in a younger subgroup.

Subjective subgroup signals require caution when the objective outcomes are null; no independent betaine effect can be isolated.

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Key findings

  • Objective measures did not show improvement.
  • Parents reported subjective improvement in a younger subgroup.
  • The recorded outcome domains were Breathing; Motor and behavioral function; Growth.
  • The result applies to the tested exposure: Folate plus betaine versus placebo; 12 months.

What this study can and cannot tell us

  • Subjective subgroup signals require caution when the objective outcomes are null; no independent betaine effect can be isolated.
  • 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: Accessible full-text extract plus indexed bibliographic data. Missing method details, funding information or participant denominators are not inferred.

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Dr. Khaled Mahmoud, MBBChCritical Care Medicine, Longevity Science & Editorial Direction Dr. Amr Yasser, PhDCardiometabolic, Musculoskeletal, Gut & Bioavailability Dr. Abdulla Mostafa, PhDCellular Senescence, Neurological, Mitochondrial & Brain-Ageing Biology