Results 151 to 160 of about 18,928 (260)

Longitudinal Clinical Progression in X‐Linked Adrenoleukodystrophy: The AMNL Scoring System

open access: yesAnnals of the Child Neurology Society, EarlyView.
ABSTRACT Objective The current clinical nomenclature for individuals with ABCD1 gene dysfunction is often uninformative. The disorder was initially described as a combination of adrenal insufficiency and leukodystrophy, leading to the widespread use of “X‐linked adrenoleukodystrophy” (ALD).
Eda G. Kabak   +7 more
wiley   +1 more source

Virtual Twin Approach Using Physiologically Based Pharmacokinetic Modeling to Support Precision Dosing of Valproic Acid in Geriatric Patients

open access: yesClinical Pharmacology &Therapeutics, EarlyView.
Personalized dosing is particularly important for drugs with narrow therapeutic indices in geriatric patients, who exhibit substantial physiological variability and limited pharmacokinetic (PK) evidence to guide individualized dose selection. Valproic acid (VPA) is an effective treatment option for bipolar disorder in older adults, yet dosing largely ...
Yoo Jin Jang   +2 more
wiley   +1 more source

<i>HSD17B4</i>-Related Disorder: Defining the Phenotype in Adult-Onset Patients. [PDF]

open access: yesNeurol Genet
Falcone GMI   +6 more
europepmc   +1 more source

Operationalizing Precision Medicine in Drug Development: Predictive Biomarkers, Companion Diagnostics, and Regulatory Pathways

open access: yesClinical Pharmacology &Therapeutics, EarlyView.
Precision medicine offers the opportunity to improve the benefit–risk profile of new therapies by prospectively identifying patients most likely to respond or least likely to experience harm; however, its systematic integration into drug development remains inconsistent outside oncology.
Ingrid Holst‐Laubjerg   +1 more
wiley   +1 more source

Seminars in epileptology: Holistic management of epilepsy in adults with intellectual development disorders

open access: yesEpileptic Disorders, EarlyView.
Abstract This seminar addresses the complexity of the management of epilepsy in adults with intellectual development disorders (IDD), advocating holistic and multidisciplinary care aligned with the learning objectives of the International League Against Epilepsy. Epilepsy is significantly more prevalent in people with IDD, presenting unique diagnostic,
Elena Fonseca   +10 more
wiley   +1 more source

Electroclinical phenotypes—genetic characterization of developmental and epileptic encephalopathies in a cohort study

open access: yesEpileptic Disorders, EarlyView.
Abstract Objective Developmental and epileptic encephalopathies (DEEs) are characterized by refractory seizures and frequently recurring epileptic activity with neurodevelopmental delay or regression that usually begin in early life. We aimed to define the relationship between electroclinical features and etiology, as well as the genotype–phenotype ...
Burcu Yaman   +7 more
wiley   +1 more source

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