The Use of Synaptic Extracellular Myo‐Inositol to Treat Developmental and Epileptic Encephalopathy
High-dose enteral myo-inositol supplementation was safely used in a patient with epileptic encephalopathy due to PLCB1 deletion, increasing CSF levels and improving seizures and brain atrophy. The hypothesized mechanism involves restoring a fetal-like state with increased membrane potential, thereby reducing neuronal firing. Based on our experience, we encourage the exploration of high-dose myo-inositol in clinical trials involving infants with severe epileptic encephalopathy.
Authors
- Susan A. Berry (ORCID: https://orcid.org/0000-0001-7970-688X)
- Annapurna Poduri (ORCID: https://orcid.org/0000-0002-7350-5136)
- Edward Yang (ORCID: https://orcid.org/0000-0002-9912-4163)
- Michael J. Bennett (ORCID: https://orcid.org/0000-0002-1889-5442)
- Sanjay P. Prabhu (ORCID: https://orcid.org/0000-0003-0871-115X)
- Didem Demirbas (ORCID: https://orcid.org/0000-0003-2865-2708)
- Robin L. Haynes (ORCID: https://orcid.org/0000-0003-4909-9585)
- E. Naomi Vos (ORCID: https://orcid.org/0000-0001-5363-9041)
- Lance H. Rodan (ORCID: https://orcid.org/0000-0002-2392-6688)
- Phillip L. Pearl (ORCID: https://orcid.org/0000-0002-6373-1068)
- M. Estela Rubio‐Gozalbo (ORCID: https://orcid.org/0000-0001-7182-4056)
- Miao He (ORCID: https://orcid.org/0000-0003-0731-6801)
- Xiaoping Huang (ORCID: https://orcid.org/0000-0002-6826-0322)
- Wanshu Qi (ORCID: https://orcid.org/0000-0003-4040-8935)
- Maria K. Lehtinen
- Jie Chen (ORCID: https://orcid.org/0000-0002-4637-672X)
Institutions
- Boston Children's Hospital (US)
- National Institutes of Health (US)
- Children's Hospital of Philadelphia (US)
- Harvard University (US)
- Maastricht University Medical Centre (NL)
- Maastricht University (NL)
- Netherlands Center for Occupational Diseases (NL)
- National Institute of Neurological Disorders and Stroke (US)
- ERN GUARD-Heart (NL)
- Boston Children's Museum (US)
- Epilepsy Foundation (AU)
- Dana-Farber/Boston Children's Cancer and Blood Disorders Center (US)
Publication Details
- Journal
- Annals of the Child Neurology Society
- Published
- 2025-08-25
- DOI
- https://doi.org/10.1002/cns3.70029
- Citations
- 1
- Primary Topic
- Epilepsy research and treatment
- Type
- article
- Field-Weighted Citation Impact
- 1.47
Funders
- Diamond Blackfan Anemia Foundation
- National Institutes of Health
- National Institute of Neurological Disorders and Stroke