Tier 2 — strong

A placebo-controlled trial of folic acid and betaine in identical twins with Angelman syndrome

Julia Han, Terry Jo Bichell, Stephanie Golden, Irina Anselm, Susan Waisbren, Carlos A Bacino, Sarika U Peters, Lynne M Bird, Virginia Kimonis
Orphanet journal of rare diseases 2019 14(1):232

Bibliography

PubMed
PMID 31640736
PubMed Central
PMC6806546
DOI
10.1186/s13023-019-1216-0
Funding
Reported funding/support: NIHU54 RR019478
Competing interests
Reported disclosure: The authors declare that they have no competing interests of this manuscript.

Study snapshot

DesignPlacebo-controlled twin study
ModelHuman participants
Sample4 boys with Angelman syndrome; two identical-twin pairs
InterventionBetaine plus folic acid versus placebo
Duration1 year
EndpointsDevelopment; Clinical function; Adverse events

What the study showed, in plain terms

Published in 2019, this source examined brain health and combination interventions. Design: Placebo-controlled twin study. Sample/model: 4 boys with Angelman syndrome; two identical-twin pairs. The exposure or intervention was Betaine plus folic acid versus placebo. Follow-up or treatment period: 1 year.

No statistically significant developmental or clinical benefit was found; no significant adverse events were reported.

Extremely small study of a nutrient combination; absence of a detected benefit or harm has wide uncertainty.

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Key findings

  • No statistically significant developmental or clinical benefit was found; no significant adverse events were reported.
  • The recorded outcome domains were Development; Clinical function; Adverse events.
  • The result applies to the tested exposure: Betaine plus folic acid versus placebo; 1 year.

What this study can and cannot tell us

  • Extremely small study of a nutrient combination; absence of a detected benefit or harm has wide uncertainty.
  • 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