Tier 2 — strong

Double-blind therapeutic trial in Angelman syndrome using betaine and folic acid

Sarika U Peters, Lynne M Bird, Virginia Kimonis, Daniel G Glaze, Lina M Shinawi, Terry Jo Bichell, Rene Barbieri-Welge, Mark Nespeca, Irina Anselm, Susan Waisbren, Erica Sanborn, Qin Sun, William E O'Brien, Arthur L Beaudet, Carlos A Bacino
American journal of medical genetics. Part A 2010 152A(8):1994-2001

Bibliography

PubMed
PMID 20635355
PubMed Central
PMC3172130
DOI
10.1002/ajmg.a.33509
Funding
Reported funding/support: National Institute of Child Health & Human Development : NICHDR01 HD037283-12 || HDNational Institute of Child Health & Human Development : NICHDR01 HD037283-11A2 || HDNational Institute of Child Health & Human Development : NICHDR01 HD037283-10 || HDNational Institute of Child Health & Human Development : NICHDR01 HD037283-09 || HDNational Institute of Child Health & Human Development : NICHDR01 HD037283-08 || HD
Competing interests
A competing-interest declaration was not available in the indexed record or accessible disclosure metadata.

Study snapshot

DesignRandomized controlled trial
ModelHuman participants
Sample48 children with Angelman syndrome
InterventionBetaine plus folic acid versus placebo
Duration1 year
EndpointsDevelopment; Clinical function; EEG and biochemistry

What the study showed, in plain terms

Published in 2010, this source examined brain health and combination interventions. Design: Randomized controlled trial. Sample/model: 48 children with Angelman syndrome. The exposure or intervention was Betaine plus folic acid versus placebo. Follow-up or treatment period: 1 year.

The combination did not significantly improve developmental or clinical outcomes compared with placebo.

Subgroup trends do not overturn the overall null comparison; results apply to the combination and this rare disorder.

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Key findings

  • The combination did not significantly improve developmental or clinical outcomes compared with placebo.
  • The recorded outcome domains were Development; Clinical function; EEG and biochemistry.
  • The result applies to the tested exposure: Betaine plus folic acid versus placebo; 1 year.

What this study can and cannot tell us

  • Subgroup trends do not overturn the overall null comparison; results apply to the combination and this rare disorder.
  • 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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Editorial review

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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