vigabatrin / Generic mfg. - LARVOL DELTA

Vigabatrin-associated brain MRI abnormalities in children: Associations with drug exposure and epilepsy etiology. (PubMed, Seizure) - "Our findings support a potential relationship between vigabatrin exposure and VABAM while highlighting the sensitivity of this association to model specification. The differing etiologic and genetic profiles observed between patients with and without VABAM raise the possibility that underlying epilepsy etiology may also influence susceptibility. Given the small number of patients with individual genetic disorders, these findings should be considered hypothesis-generating and warrant validation in larger cohorts."

Journal • CNS Disorders • Epilepsy • Genetic Disorders

Long-term developmental outcome in infantile epileptic spasms syndrome after high-dose prednisolone and vigabatrin treatment. (PubMed, Epilepsia Open) - "Infantile epileptic spasms syndrome (IESS) is a severe epilepsy of infancy that often leads to lasting developmental problems. In this study of 236 children with IESS treated with a stepwise vigabatrin and prednisolone protocol, developmental outcome at age 3 was mainly associated with the underlying cause of IESS and response to treatment. Children without an identified underlying cause and those who responded to treatment had better developmental outcomes than children with a structural or genetic cause or those who did not respond. These findings emphasize the importance of careful evaluation and individualized treatment in children with IESS."

Journal • CNS Disorders • Epilepsy

Pharmacokinetics of Loading Dose Vigabatrin Administered via Enteral Tube in Participants With Post-Anoxic Status Epilepticus. (PubMed, Pharmacotherapy) - "This pioneer vigabatrin PK study in critically ill participants with PASE demonstrated absorption regardless of challenges such as enteral delivery type, co-administration of gastric pH modulators, vasopressors, and anesthetics. Notably, prolonged elimination half-lives and increased exposures were observed."

Clinical • Journal • PK/PD data • Anesthesia • Cardiovascular • CNS Disorders • Epilepsy • Vascular Neurology

A case of bilateral open-lip schizencephaly with West syndrome due to variant of PAFAH1B1 gene and literature review (PubMed, Zhonghua Yi Xue Yi Chuan Xue Za Zhi) - "PAFAH1B1 gene variants are an important cause for cortical malformations, including schizencephaly, and may lead to secondary West syndrome. This study has systematically summarized the genotypic and phenotypic features of bilateral open-lip schizencephaly complicated by West syndrome associated with a frameshift variant of the PAFAH1B1 gene. For infants with epileptic spasms or early-onset epilepsy accompanied by structural brain abnormalities, early genetic evaluation should be performed, and antiseizure treatment should be integrated with neurodevelopmental rehabilitation to facilitate long-term management."

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

RFX3 Pathogenic Variants as a Rare Cause of Infantile Epileptic Spasms Syndrome. (PubMed, Int J Mol Sci) - "The patient developed clusters of extensor spasms associated with eye deviation and achieved complete remission within two weeks following vigabatrin and ACTH therapy, remaining seizure-free thereafter...This case expands the clinical spectrum associated with RFX3 variants, supporting a potential role in IESS and early neurodevelopmental disruption. It highlights the relevance of including RFX3 in the genetic evaluation of patients with IESS and co-occurring neurodevelopmental disorders."

Journal • ADHD (Impulsive Aggression) • Attention Deficit Hyperactivity Disorder • Autism Spectrum Disorder • CNS Disorders • Developmental Disorders • Epilepsy • Genetic Disorders • Psychiatry • Strabismus

Sequential surrogate optimization for supercritical fluid extraction - Supercritical fluid chromatography - Tandem mass spectrometry method development. (PubMed, Anal Chim Acta) - "Analytes, specifically reserpine, vigabatrin, hydromorphone, and hydrocodone, covering diverse physicochemical profiles, were optimized sequentially, guided by a molecular similarity metric, to broaden the design space. Sensitivity analysis over 62.5-1000 ng mL-1 yielded detection limits/quantification limits of 6.9/23.0 and 2.4/8.1 ng mL-1 for hydrocodone and reserpine, respectively. Sequential optimization enhanced sensitivity by 15%, demonstrating the scalability and transferability of the surrogate optimization framework for developing SFE-SFC methods."

Journal

Breaking a translational barrier in the development of a GABA aminotransferase inactivator (ACS-Fall 2026) - "CPP-115 has demonstrated strong therapeutic potential as a next-generation inactivator of GABA aminotransferase (GABA-AT) with markedly improved potency and safety compared to the only clinically available GABA-AT inactivator, vigabatrin. Based on this insight, a next-generation inactivator with substantially improved selectivity was developed and is now undergoing preclinical evaluation as a potentially safer candidate. Future directions with this molecule include proteomic profiling to systematically validate the selectivity across homologous enzymes and the development of activity-based probes, offering potential avenues for both therapeutic advancement and translational applications."

CNS Disorders • Epilepsy

An Open Label Study of Alcohol Withdrawal Syndrome in the Outpatient Setting Using Vigabatrin (ANZCTR) - P2 | N=50 | Recruiting | Sponsor: St Vincents Hospital Melbourne

New P2 trial

Morin, a bioflavonoid from a traditional medicinal plant, attenuates inflammasome-associated neuroinflammation in drug-resistant epilepsy. (PubMed, Eur J Pharmacol) - "Drug resistance was established through chronic co-administration and confirmed by non-responsiveness to carbamazepine and valproate. Morin (20 and 40 mg/kg), alone or in combination with valproate, was evaluated against vigabatrin for its impact on seizure severity, neurochemistry, and neuropathology...Additionally, morin-based treatments reduced caspase-1, IL-1β, and NQO1 levels in the hippocampus and cortex, improved neuronal architecture, and attenuated microglial and astroglial activation. These findings suggest that morin may be a promising adjunctive candidate for DRE by modulating seizure severity, excitatory-inhibitory imbalance, oxidative stress, and inflammation associated with inflammasome-linked signaling."

Journal • CNS Disorders • Epilepsy • Inflammation • IL1B • NQO1

Balancing Risk and Response: ACTH Therapy for Infantile Spasms in Citrullinemia Type I (SSIEM 2026) - "First-line treatment includes corticosteroids (CS), such as adrenocorticotropic hormone (ACTH), followed by vigabatrin...Intensive monitoring included frequent ammonia levels and daily plasma amino acids, with adjustments to diet (Cyclinex-1, breast milk, arginine) and sodium phenylbutyrate... Adrenocorticotropin can be safely administered in citrullinemia type I with intensive metabolic support and multidisciplinary care. This case challenges conventional contraindications and underscores the importance of individualized risk–benefit assessment in treating IS in UCD patients."

CNS Disorders • Endocrine Disorders • Epilepsy • Metabolic Disorders • Nephrology • ASS1

Impact of vigabatrin on risk of relapse of infantile spasms. (PubMed, Epilepsia Open) - "Infantile Epileptic Spasms Syndrome is a serious seizure disorder of infancy that can recur even after successful treatment with vigabatrin. In this study of 164 infants, we found that relapse was most common among children with prior relapses, other seizure types, or developmental delays. Infants treated with moderate vigabatrin doses (100-149 mg/kg/day) had the lowest relapse rates. These findings may help clinicians identify which infants face the highest risk and tailor treatment accordingly."

Journal • Cardiovascular • CNS Disorders • Developmental Disorders • Epilepsy

Management of Infantile Epileptic Spasms Syndrome: A survey of US pediatric hospitals. (PubMed, Epilepsia Open) - "Infantile Epileptic Spasms Syndrome requires prompt diagnosis and initiation of appropriate treatment to improve developmental outcomes. In this survey of tertiary pediatric centers in the United States, we found areas of consensus (use of overnight video EEG at initial diagnosis, use of high-dose prednisolone as initial treatment, preference for sequential as opposed to combination therapy, use of the 2021 BASED score for EEG evaluation, and the early consideration of epilepsy surgery) and variability (type of genetic testing for diagnosis, choice of second and third treatments, use of antiseizure medications for persistently epileptic EEG) in management of these patients."

Journal • Cardiovascular • CNS Disorders • Epilepsy • Pediatrics

GRIN1-related epilepsy in a neonate with response to memantine and vigabatrin. (PubMed, Ann Child Neurol Soc) - "Patients with gain-of-function GRIN1 mutations who present with the phenotype of DEE with extensive bilateral polymicrogyria may benefit from a trial of NMDA-receptor antagonist therapy and vigabatrin. Further research is warranted to better understand this markedly pharmacoresistant condition and to investigate targeted therapies in GRIN1 DEE."

Journal • Cardiovascular • CNS Disorders • Critical care • Epilepsy • Movement Disorders

Demographic characteristics and clinical presentation of infants with infantile epileptic spasms syndrome and their response to therapy: Data from Sri Lanka Infantile Spasms Registry. (PubMed, Ann Child Neurol Soc) - "Response to treatment for standard (adrenal corticotropic hormone, prednisolone, or vigabatrin) versus nonstandard medications was evaluated at two and six weeks...While both standard therapies led to positive outcomes, oral prednisolone produced the best therapeutic response...Although most patients received a recommended first-line therapy (most often prednisolone), a sizable number initially received nonstandard therapy. Our data illustrate the challenges in the management of IESS in a resource-limited environment."

Journal • CNS Disorders • Epilepsy • Pediatrics

Neonatal Seizures in Tuberous Sclerosis Complex: A Case Series. (PubMed, Ann Child Neurol Soc) - "All patients with neonatal seizures had TSC2 and developed drug-resistant epilepsy, and two of four developed infantile spasms despite intensive early medical management, including vigabatrin...They have not developed epilepsy and have had milder neurodevelopmental impairment.In conclusion, data from our center suggest that neonatal seizures may be common in TSC, especially in high-risk infants with TSC2 with highly epileptogenic lesions. With recent preliminary data about safety and efficacy of mTOR inhibitor treatment in infants with TSC, early EEG monitoring should be considered."

Journal • CNS Disorders • Epilepsy • TSC1 • TSC2

Vigabatrin-Induced Retinal Toxicity: Unraveling the Gamma-Aminobutyrate-Vitamin B6 Axis and Metabolic Crosstalk. (PubMed, J Appl Toxicol) - "Understanding this complex interplay between GABA-vitamin B6 axis and metabolic crosstalk offers opportunities for safer therapeutic approaches and potential protective strategies for patients undergoing long-term VGB therapy. Future research should focus on mechanistic studies, pharmacokinetic profiling, genetic studies, and longitudinal clinical investigations to maximize both seizure control and retinal safety."

Journal • Review • CNS Disorders • Epilepsy • Metabolic Disorders

Everolimus initiated in the neonatal period for refractory seizures associated with tuberous sclerosis complex: long-term clinical outcome. (PubMed, Epilepsy Behav Rep) - "Despite treatment with phenobarbital, midazolam, and vigabatrin, seizures remained uncontrolled. Early developmental assessment at 12 months showed cognitive, language, and motor scores within the lower range of normal. This case suggests that early initiation of everolimus may represent a feasible adjunctive treatment option for selected neonates with TSC-associated refractory seizures."

Clinical data • Journal • CNS Disorders • Epilepsy • Oncology

Seven-Year Longitudinal Respiratory Morbidity in Ohtahara Syndrome: A Case Report Emphasizing Integrated Airway and Seizure Care in a Resource-Limited Setting. (PubMed, Case Rep Pediatr) - "Despite combination antiseizure therapy (phenobarbital, levetiracetam, vigabatrin, and clonazepam), she had breakthrough events and severe developmental impairment. In OS, respiratory complications, often driven by aspiration risk and impaired airway clearance, can become a dominant source of morbidity and should be addressed with the same priority as antiseizure therapy. We highlight practical pillars for long-term care: proactive infection prevention and early treatment pathways; aspiration-risk assessment with feeding/swallow support when feasible; airway-clearance planning with bronchodilator trials during illnesses; judicious corticosteroid use; and caregiver education with explicit thresholds for escalation."

Journal • CNS Disorders • Epilepsy • Infectious Disease • Pneumonia • Respiratory Diseases

Management of Infantile Epileptic Spasms Syndrome: Cross-Canada Comparison of Protocols and Practices for the Management of Infantile Spasms (CNSF 2026) - "Vigabatrin and high dose steroids are first line, but there is a lack of guidance on appropriate dosage and duration... There is consistent use of the recommended first line medications for infantile spasms management across Canada, but there is variability in order, dosing, monitoring, and in the management of refractory patients. By understanding more about practice across Canada, we can work towards developing a national consensus approach to the treatment of infantile spasms."

Clinical • CNS Disorders • Epilepsy

Vigabatrin therapy for infantile epileptic spasms syndrome with periventricular leukomalacia. (PubMed, Brain Dev) - "Vigabatrin was effective for treating 42.8% of our patients with both IESS and PVL. Vigabatrin was well tolerated, with only a mild effect observed in patients with IESS and PVL. Some treatment-related ERG abnormalities were normalized after vigabatrin was reduced or stopped."

Journal • Cardiovascular • CNS Disorders • Epilepsy • Ophthalmology • Respiratory Diseases • Vascular Neurology

Treatment history as early indicators of short-term response to ACTH therapy in infantile epileptic spasms syndrome: a propensity score matching analysis. (PubMed, Neurol Res Pract) - "The addition of new ASMs and frequent dose adjustments during ACTH therapy might reflect early clinical concern for poor response and should prompt reassessment of treatment strategy. Recognizing these signals may facilitate timely clinical intervention."

Journal • CNS Disorders • Epilepsy

Clinical characteristics of six cases of neurofibromatosis type 1-associated infantile epileptic spasms syndrome (PubMed, Zhongguo Dang Dai Er Ke Za Zhi) - "Children with NF1 and IESS commonly present with the typical triad of IESS. Some patients show NF1-related T2 hyperintense lesions in the basal ganglia on brain magnetic resonance imaging. Standard first-line treatments often control spasms effectively, but neurodevelopmental outcomes require long-term monitoring."

Journal • Cardiovascular • CNS Disorders • Developmental Disorders • Epilepsy • Genetic Disorders • Neurofibromatosis • Solid Tumor • NF1

Vigabatrin-related MRI changes and tapering strategy in hemispherotomy for infantile spasms: illustrative case. (PubMed, J Neurosurg Case Lessons) - "This case demonstrates that vigabatrin-related MRI abnormalities can closely mimic serious metabolic or vascular conditions, but are reversible and should not delay surgery when pharmacoresistance is established. Postoperative tapering can be safely implemented once seizure control is achieved, supporting timely intervention and avoiding unnecessary investigations. https://thejns.org/doi/10.3171/CASE26183."

Journal • Cardiovascular • CNS Disorders • Epilepsy

Seizure-induced apnea causes sudden death independent of cortical spike wave discharge in mouse models of Tuberous Sclerosis Complex. (PubMed, Neurobiol Dis) - "In addition, Vigabatrin-treated mice exhibited cardiac signals that declined hours before SUDEP. These findings underscore the need for SUDEP-specific interventions beyond seizure suppression."

Journal • Preclinical • CNS Disorders • Epilepsy

Late-onset epileptic spasms: presentation, aetiology and outcome. (PubMed, Brain Commun) - "More children with unilateral structural-malformative aetiologies achieved seizure freedom than other aetiologies, most commonly following surgery but occasionally following treatment with vigabatrin or clobazam. This study highlights the importance of prompt recognition of epileptic spasms in older children for improved seizure and developmental outcomes. Brain malformations and insults are the predominant causes of LOES, and when unilateral, respond best to epilepsy surgery."

Journal • Cardiovascular • CNS Disorders • Cognitive Disorders • Epilepsy • Hematological Malignancies • Infectious Disease • Leukemia • Oncology • Solid Tumor