vigabatrin / Generic mfg. - LARVOL DELTA

Long-term outcome in children with infantile epileptic spasms syndrome: a multicenter retrospective study in Korea. (PubMed, Clin Exp Pediatr) - "Vigabatrin was prescribed to 93.9% of the patients, suggesting that it was the mainstay of treatment...The IESS imposes a substantial burden on affected children and their families and often leads to chronic epilepsy and impaired cognitive function. Consensus diagnostic and treatment guidelines tailored to the Korean clinical practice are necessary to ensure early diagnosis and timely treatment."

Clinical • Journal • Retrospective data • CNS Disorders • Developmental Disorders • Epilepsy • Genetic Disorders • Mental Retardation

Long term safety and efficacy of vigabatrin in Lennox-Gastuat syndrome: a long way to go. (PubMed, J Neurol Sci) - No abstract available

Journal • CNS Disorders

De novo variant of the KLF7 gene and early and degenerative infantile epileptic encephalopathy: description of a case. (HMGC 2026) - "At six months, he was diagnosed with early-onset infantile epileptic encephalopathy resistant to medication (failure of topiramate, vigabatrin, valproate, and a ketogenic diet). These cases support the involvement of KLF7 in epileptic encephalopathies, particularly when a variation affects the C-terminal domain, suggesting a region critical for gene function. The observed clinical and radiological evolution highlights a neurodevelopmental impairment that is still poorly described for KLF7."

Clinical • Cardiovascular • Cerebral Palsy • CNS Disorders • Developmental Disorders • Epilepsy

Does First-Line Treatment Impact Outcomes in Trisomy 21-Associated Infantile Epileptic Spasms Syndrome? A Multicenter North American Analysis. (PubMed, Pediatr Neurol) - "Children with T21-associated IESS were approximately two times more likely to achieve electroclinical remission with hormone therapy as first treatment compared to vigabatrin, although this difference did not reach statistical significance. The median time to remission was significantly shorter in children who received hormone therapy as their first treatment compared to those treated with vigabatrin. The initial treatment did not impact long-term clinical outcomes, such as ongoing epilepsy or ASD."

Journal • Autism Spectrum Disorder • CNS Disorders • Epilepsy • Genetic Disorders • Pediatrics

Short-term vigabatrin is safe and effective in Lennox-Gastaut syndrome. (PubMed, J Neurol Sci) - No abstract available

Journal • CNS Disorders • Epilepsy

Comment on "safety and efficacy of vigabatrin add on compared to placebo in Lennox-Gastaut syndrome (LennoVig): A single center randomized double-blind placebo-controlled trial". (PubMed, J Neurol Sci) - No abstract available

Journal • CNS Disorders • Epilepsy

Combined oxidative phosphorylation deficiency 35 due to TRIT1 variants: Novel phenotypes and ketogenic diet utilization (ACMG 2026) - "Current antiseizure and adjunctive medications include Onfi, Vimpat, leucovorin, Keppra, levocarnitine, and vigabatrin. Finally, progressive cerebral and cerebellar atrophy were demonstrated on repeat brain MRI, which has not previously been reported and may indicate utility of repeated brain imaging throughout disease course. Conclusion We describe a case of COXPD35 that increases the limited clinical understanding of disease spectrum, cardiac and brain abnormality phenotypes, and ketogenic diet for management of epileptic encephalopathy."

Cardiomyopathy • Cardiovascular • CNS Disorders • Developmental Disorders • Dystonia • Epilepsy • Mental Retardation • Movement Disorders • Ocular Inflammation • Ophthalmology • Rare Diseases • Strabismus • BCL11B

Whole-Genome Sequencing Reveals a Novel CACNA1B Missense Variant in a Child with Infantile Spasms and Neurodevelopmental Delay (ACMG 2026) - "Treatment and Management Infantile spasms were initially treated with prednisone and later with vigabatrin, leading to seizure resolution. Conclusion Whole-genome sequencing and periodic reanalysis were instrumental in identifying a candidate CACNA1B variant in a child with an undiagnosed neurodevelopmental disorder. This finding will likely contribute to the evolving understanding of calcium-channelopathies and highlights the ongoing need for integrative genomic, structural, and functional approaches to establish variant pathogenicity and improve diagnostic yield in rare disease genomics."

Clinical • Neurodevelopmental • Whole genome sequencing • Cardiovascular • CNS Disorders • Developmental Disorders • Epilepsy • Gynecology • Hypertension • Rare Diseases • Sleep Disorder • Strabismus • CACNA1B • CAV2

Unravelling GABA Dysfunction in Autism: Pathophysiological Insights and Emerging Treatments. (PubMed, Int J Dev Neurosci) - "Among these, GABAB receptor agonists, including arbaclofen and baclofen, show the most promise in improving social behaviour and reducing core ASD symptoms. Conversely, some agents that elevate GABA levels, such as vigabatrin and valproic acid, may exacerbate ASD-like features under certain conditions. Collectively, the data suggest that targeted modulation of GABAergic pathways, particularly GABAB receptor signalling, offers a viable avenue for therapeutic intervention in ASD. However, further mechanistic studies and well-designed clinical trials are required to elucidate the optimal strategies for harnessing GABA modulation in ASD management."

Journal • Review • Autism Spectrum Disorder • Genetic Disorders

Vigabatrin and the Pediatric Epilepsy Research Foundation® (AAN 2026) - No abstract available

Clinical • CNS Disorders • Epilepsy • Pediatrics

Molecular Basis of GABA Aminotransferase Inhibition in Epilepsy: Structure, Mechanisms, and Drug Development. (PubMed, Curr Issues Mol Biol) - "We discuss the structure-function relationships of GABA-T, the pharmacology of vigabatrin and experimental inhibitors, clinical efficacy across various epilepsy syndromes, adverse effect profiles, and future directions including novel inhibitors with improved safety profiles. Understanding the role of GABA-T in epilepsy pathophysiology and the therapeutic potential of its inhibitors provides insights into rational drug design and personalized treatment strategies for epilepsy management."

Journal • Review • CNS Disorders • Epilepsy

Targeting CRHR1 Signaling in Experimental Infantile Epileptic Spasms Syndrome: Evidence for Route-Dependent Efficacy. (PubMed, Children (Basel)) - "These findings demonstrate that CRHR1 blockade modifies experimental spasms in a route- and drug-specific manner and implicates discrete hypothalamic circuits, particularly those including the arcuate nucleus, in spasm generation. The divergent systemic responses between CP376395 and SN003 likely reflect differences in CRHR1 engagement (competitive and non-competitive antagonism, respectively) as well as differences in binding properties that may include differential network interactions beyond local CRH signaling or duration of receptor occupancy. In conclusion, SN003 may be a better option than CP376395 for further development as a CRHR1-targeted therapy pending additional pharmacokinetic/pharmacodynamic studies. Further work should explore dosing paradigms of CP376395 to determine if a therapeutic range for CP376395 exists."

Journal • CNS Disorders • Epilepsy

The global research hotspots and future trends of infantile epileptic spasms syndrome: A bibliometric analysis of trends and themes. (PubMed, Brain Dev) - "The findings highlight current hotspots spanning genetic mechanisms, therapeutic strategies, epidemiological patterns, clinical practices, and neuroimaging. Future research should optimize treatments, improve diagnostics, and address developmental impacts."

Journal • Cardiovascular • CNS Disorders • Developmental Disorders • Epilepsy • Mental Retardation

Etiology of Infantile Epileptic Spasms Syndrome and Clinical Response With Vigabatrin as the First Treatment. (PubMed, Pediatr Neurol) - "The etiology of IESS was identified in 75%. VGB was most effective as first-line, short-term treatment in TSC patients, and one-year treatment in children with normal development at diagnosis of IESS."

Journal • CNS Disorders • Developmental Disorders • Epilepsy • TSC1

Tuberous sclerosis presents with self-injurious behavior as an adverse event of Vigabatrin. (PubMed, Indian J Psychiatry) - No abstract available

Adverse events • Journal

Safety and efficacy of vigabatrin add on compared to placebo in Lennox-Gastaut syndrome (LennoVig): A single center randomized double-blind placebo-controlled trial. (PubMed, J Neurol Sci) - "In LGS, vigabatrin is effective in reducing drop attacks, and focal, myoclonic and atypical absence seizures along with improvement in the CGI. Clinical Trial Registry of India, Indian Council of Medical Research (CTRI/2022/12/048517)."

Journal • Absence Seizure Disorder • Anesthesia • CNS Disorders • Epilepsy

Bayesian-Based Pharmacokinetic Framework Integrated with Therapeutic Drug Monitoring for Assessing Adherence to Antiseizure Medications: A Clinical Trial Simulation Study. (PubMed, J Med Internet Res) - "This study proposed an innovative Bayesian-based pharmacokinetic (PK) framework integrated with TDM data to address the above limitations, with a focus on 14 widely prescribed ASMs, including brivaracetam, carbamazepine, clobazam, eslicarbazepine acetate, lacosamide, lamotrigine, levetiracetam, oxcarbazepine, perampanel, phenobarbital, topiramate, valproic acid, vigabatrin, and zonisamide. This study establishes a Bayesian-based PK approach to enhance the assessment of ASMs adherence. This approach facilitates a paradigm shift from population-based management to patient-specific adherence profiling, offering a practical methodology for the precise evaluation of medication-taking behaviors."

Journal • PK/PD data • CNS Disorders • Epilepsy

Anti-seizure potential of J4, an equilibrative nucleoside transporter 1 inhibitor, in a mouse model of tuberous sclerosis complex in response to pentylenetetrazol. (PubMed, Cell Biosci) - "Vigabatrin, which has been used as the first-line therapy for infantile spasms in TSC, has demonstrated to delay the onset and lower the overall incidence of seizures in infants with TSC when it was used as a preventive treatment...We also found that J4 increased the expression of GluR2, inhibited the astrogliosis and microgliosis, and eventually prevented the neuronal cell loss due to the excitotoxicity. The present study provides a new alternative therapeutic concept for pretreating TSC-related epilepsy before the epileptogenesis process."

Journal • Preclinical • CNS Disorders • Developmental Disorders • Epilepsy • Psychiatry • SLC29A1 • TSC1 • TSC2

The Therapeutic Role of Physical Activity in Epilepsy: Potential Mechanisms and Clinical Implications. (PubMed, Dev Neurobiol) - "Anti-inflammatory strategies, encompassing pharmacologic agents such as glucocorticoids, adrenocorticotropic hormone, and vigabatrin, as well as dietary approaches like the ketogenic diet, have demonstrated reductions in seizure frequency and inflammatory signaling...Collectively, these findings support the potential of integrating exercise-based interventions to mitigate neuroinflammation and seizure burden, particularly in aging populations. Further research is needed to clarify underlying mechanisms and optimize protocols for clinical translation."

Journal • Review • Asthma • CNS Disorders • Depression • Epilepsy • Immunology • Inflammation • Mood Disorders • Oncology • Psychiatry • Pulmonary Disease • Respiratory Diseases • IL1B • IL6 • TNFA

Infantile Spasms (West Syndrome): Integrating Genetic, Neurotrophic, and Hormonal Mechanisms Toward Precision Therapy. (PubMed, Medicina (Kaunas)) - "Early treatment with ACTH or high dose prednisolone, with or without vigabatrin, was consistently associated with higher electroclinical remission and better developmental outcome. Everolimus and related mTOR inhibitors showed benefit in TSC-associated ISs, while agents directed at NMDA receptors or KCNQ channels are emerging for genotype defined subgroups. ISs should be approached as a heterogeneous but mechanistically convergent disorder in which rapid diagnosis, parallel genetic testing, and early disease modifying therapy improve prognosis. Integration of molecular profiling with standardized outcome monitoring is likely to move management from symptomatic seizure control to pathway-specific intervention."

Journal • Review • Cardiovascular • CNS Disorders • Epilepsy • BDNF • GRIN2A • GRIN2B • IGF1 • XBP1

Real-world disproportionality analysis of sleep disturbances associated with antiseizure medications in epilepsy: a pharmacovigilance study. (PubMed, Front Pharmacol) - "Significant signals were detected for sodium channel blockers eslicarbazepine, stiripentol, and cenobamate, as well as for mechanistically diverse agents vigabatrin, pregabalin, brivaracetam, and cannabidiol. The distinct disproportionality reporting profiles identified for some agents, which differ from prior evidence, necessitate careful clinical interpretation. Overall, this study elucidates the complex sleep safety profiles of ASMs, offering evidence to support more informed drug selection and monitoring in practice."

Adverse events • Journal • Real-world evidence • CNS Disorders • Epilepsy • Insomnia • Sleep Disorder

Vigabatrin-Associated Brain Magnetic Resonance Imaging Abnormalities in Two Children With WW domain-containing oxidoreductase-Related Epileptic Encephalopathy Syndrome. (PubMed, Pediatr Neurol) - "Brain magnetic resonance imaging during vigabatrin treatment revealed new symmetrical signal changes in the globus pallidi and thalami consistent with VABAM. Further research is warranted to investigate whether children with genetic epilepsy related to the GABAergic pathway or delayed myelination are more susceptible to VABAM."

Journal • CNS Disorders • Developmental Disorders • Epilepsy • Gastrointestinal Disorder

Infantile epileptic spasms syndrome: Mechanisms and therapeutic approaches. (PubMed, Neurotherapeutics) - "epileptic encephalopathy with unique clinical and electrographic features, including seizure semiology (spasms), numerous and diverse etiologies spanning structural, genetic and metabolic causes, characteristic interictal (hypsarrhythmia) and ictal (electrodecrement) electroencephalogram (EEG) patterns, and responsiveness to "standard" pharmacological therapies (adrenocorticotrophic hormone, high-dose corticosteroids, vigabatrin) that are not commonly used in other epilepsy syndromes. Despite these long-recognized clinical features and laboratory investigations using a multiplicity of animal models with different epileptogenic mechanisms, the neurobiological underpinnings of IESS remain poorly understood, hampering the development of alternative treatments. This commentary discusses three aspects of IESS intended to raise fundamental clinical and mechanistic issues to afford greater understanding of the syndrome - nomenclature, EEG findings, and selected..."

Journal • Review • Cardiovascular • CNS Disorders • Epilepsy

Reassessing vigabatrin in infantile spasms: clinical risks, mechanistic insights, and future directions. (PubMed, Pediatr Res) - No abstract available

Journal • CNS Disorders • Epilepsy

Genetic Etiology of Infantile Spasms in Peruvian Children: A Multicenter Study. (PubMed, J Child Neurol) - "Vigabatrin was the most frequently used control medication (54.55%, n = 30)...A wide range of genetic variants was found in one-third of the cases. Prompt genetic identification in IESS is recommended to optimize treatment and improve the prognosis."

Clinical • Journal • Cardiovascular • CNS Disorders • Developmental Disorders • Epilepsy • Pediatrics