Results 191 to 200 of about 21,502,425 (295)

Gene Therapy for Amino Acid Decarboxylase Deficiency: Clinical and Imaging Outcomes in a French Cohort

open access: yesMovement Disorders, EarlyView.
Abstract Background Intracerebral gene therapy is effective for amino acid decarboxylase (AADC) deficiency, but relationships between anatomical putaminal coverage, metabolic dynamics, and clinical recovery remain poorly understood. Objectives Assess safety, long‐term efficacy, and clinical–radiological correlations in a genetically diverse European ...
Clément Dunoyer   +27 more
wiley   +1 more source

Increased GH/IGF-I Axis Activity Relates to Lower Hepatic Lipids and Phosphor Metabolism.

open access: yesJ Clin Endocrinol Metab, 2023
Fellinger P   +15 more
europepmc   +1 more source

Key Interventions in Friedreich's Ataxia and Their Impact on Patient Outcomes: A Systematic Review

open access: yesMovement Disorders, EarlyView.
Abstract Friedreich's ataxia (FA) is a rare neurodegenerative disease with multisystemic symptoms that requires multidisciplinary care. This systematic review summarizes available pharmacological and nonpharmacological interventions, their outcomes, and alignment with patient‐centered care domains, as well as their impact on these domains.
Dorota Sarwinska   +6 more
wiley   +1 more source

High‐Fat Diet Exacerbates Neuropathology in a Transgenic Mouse Model of Multiple System Atrophy

open access: yesMovement Disorders, EarlyView.
Abstract Background Multiple system atrophy (MSA) is a rare and devastating neurodegenerative disorder. Accumulating clinical and preclinical evidence suggests that diabetes and insulin resistance may adversely influence MSA pathophysiology. Objective We investigated the potential association between diabetes, impaired glucose homeostasis, and MSA ...
Marie‐Laure Arotcarena   +7 more
wiley   +1 more source

Loss-of-Function GHSR Variants Are Associated With Short Stature and Low IGF-I.

open access: yesJ Clin Endocrinol Metab
Punt LD   +28 more
europepmc   +1 more source

Redefining CHI3L1: Therapeutic Opportunities at the Crossroads of Immune Suppression and Disease Progression

open access: yesMedicinal Research Reviews, EarlyView.
ABSTRACT Chitinase‐3‐like‐1 (CHI3L1, also known as YKL‐40) has been recognized as a biomarker of inflammation and tissue remodeling and has now emerged as a pseudoenzymatic immune checkpoint. Recent structural, immunological, and translational studies redefine it as an active regulator of immune suppression rather than a passive disease marker. Despite
Kirti Upmanyu   +2 more
wiley   +1 more source

Implication of Pappalysins and Stanniocalcins in the Bioavailability of IGF-I in Children With Type 1 Diabetes Mellitus. [PDF]

open access: yesJ Endocr Soc
Güemes M   +6 more
europepmc   +1 more source

Senotherapeutics for Knee Osteoarthritis

open access: yesMedicinal Research Reviews, EarlyView.
ABSTRACT Osteoarthritis (OA) is a chronic disease that imposes a significant economic burden and deteriorates quality of life. Nevertheless, current therapeutic options for OA are limited to symptomatic remedies. As such, there is a high interest in novel methods for treating or preventing OA.
Ezgi Duman   +6 more
wiley   +1 more source

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