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Exit interviews from two randomised placebo-controlled phase 3 studies with caregivers of young children with autism spectrum disorder.
Front Child Adolesc Psychiatry
June 2024
IM Franchise Department, Les Laboratoires SERVIER, Global Value, Access & Pricing, Suresnes, France.
Introduction: Autism spectrum disorder (ASD) is characterised by difficulty with social communication and restricted, repetitive patterns of behaviour. This study aimed to improve understanding of the ASD patient experience with the treatment (bumetanide) regarding the changes in core symptoms and to assess changes considered as meaningful. To achieve this, qualitative interviews were conducted with caregivers of patients in two phase 3 clinical trials (NCT03715153; NCT03715166) of a novel ASD treatment.
View Article and Find Full Text PDFWONOEP appraisal: Targeted therapy development for early onset epilepsies.
Epilepsia
November 2024
Saul R. Korey Department of Neurology, Isabelle Rapin Division of Child Neurology, Laboratory of Developmental Epilepsy, Dominick P. Purpura Department of Neuroscience, Albert Einstein College of Medicine, Bronx, New York, USA.
The early onset epilepsies encompass a heterogeneous group of disorders, some of which result in drug-resistant seizures, developmental delay, psychiatric comorbidities, and sudden death. Advancement in the widespread use of targeted gene panels as well as genome and exome sequencing has facilitated the identification of different causative genes in a subset of these patients. The ability to recognize the genetic basis of early onset epilepsies continues to improve, with de novo coding variants accounting for most of the genetic etiologies identified.
View Article and Find Full Text PDFThe pediatric psychopharmacology of autism spectrum disorder: A systematic review - Part II: The future.
Prog Neuropsychopharmacol Biol Psychiatry
January 2025
Department of Public Health and Pediatric Sciences, Section of Child and Adolescent Neuropsychiatry, University of Turin, Turin, Italy.
Part I of this systematic review summarized the state-of-the-art of pediatric psychopharmacology for Autism Spectrum Disorder (ASD), a severe and lifelong neurodevelopmental disorder. The purpose of this Part II follow-up article is to provide a systematic overview of the experimental psychopharmacology of ASD. To this aim, we have first identified in the Clinicaltrials.
View Article and Find Full Text PDFTorsemide in Edema Associated with Hepatic Impairment.
J Assoc Physicians India
September 2024
Country Head, Medical Affairs, Cipla Ltd., Mumbai, Maharashtra, India.
Hepatic edema is caused by decreased hepatic protein synthesis, a consequence of decompensated liver cirrhosis. Fluid accumulation occurs when there is an increase in hydrostatic pressure in the hepatic sinusoids and splanchnic capillaries, as well as low albumin. The first-line treatment for cirrhosis-related ascites is an aldosterone antagonist (spironolactone); however, in severe and recurring ascites, a combination of aldosterone antagonists and loop diuretics (torsemide, furosemide, and bumetanide) is preferable.
View Article and Find Full Text PDFEmerging Role of Low-dose Loop Diuretics in the Management of Heart Failure Patients to Maintain Euvolemia.
J Assoc Physicians India
September 2024
Cluster Medical Head, Medical Affairs, Cipla Ltd., Mumbai, Maharashtra, India.
The current scenario of employing loop diuretics in combination with guideline-directed medical therapy (GDMT) demonstrates a comprehensive approach to improving clinical outcomes in individuals with heart failure (HF). GDMT uses four types of drugs: angiotensin receptor-neprilysin inhibitors (ARNIs), beta-blockers (BBs), mineralocorticoid receptor antagonists (MRAs), and sodium-glucose cotransporter-2 inhibitors (SGLT2i). Torsemide, furosemide, and bumetanide are common loop diuretics used to control fluid overload in HF and provide symptomatic relief.
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