Results 191 to 200 of about 14,157,417 (290)

Observations on an Open‐Label Phase 1/2 Dopamine Gene Therapy Trial (OXB‐102/Axo‐Lenti‐PD) in People with Parkinson's Disease

open access: yesMovement Disorders, EarlyView.
Abstract Background SUNRISE‐PD was a dose‐escalating, phase 1/2 study investigating a second‐generation lentiviral vector gene therapy delivering the genes for dopamine synthesis (OXB‐102) to treat Parkinson's disease (PD). The trial was prematurely terminated due to insolvency of the sponsor.
Simon Rowe   +19 more
wiley   +1 more source

Maternal Anemia and Pediatric Neurodevelopment in Children from Mothers Exposed to Mixed Heavy Metals in Suriname. [PDF]

open access: yesJ Xenobiot
Gokoel AR   +6 more
europepmc   +1 more source

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

Optimizing Research Operations and Resource Utilization in ALS Care: Insights From the Tofersen Antisense Oligonucleotide Expanded Access Protocol

open access: yesMuscle &Nerve, EarlyView.
ABSTRACT Introduction/Aims Tofersen is a gene‐targeted therapy for individuals with superoxide dismutase 1 (SOD1) (+) amyotrophic lateral sclerosis (ALS). Prior to U.S. Food and Drug Administration (FDA) approval, tofersen was made available through expanded access protocol.
Alison Wheeler   +30 more
wiley   +1 more source

[Hyperuricaemia as a cardiovascular risk factor]. [PDF]

open access: yesAten Primaria
Álvarez Lozano B   +3 more
europepmc   +1 more source

[Impulse control disorder: patients with pituitary adenomas and dopaminergic agonists]. [PDF]

open access: yesRev Med Inst Mex Seguro Soc
Barreras-Ruíz A   +4 more
europepmc   +1 more source

One‐Year Functional Satisfaction and Quality of Life Outcomes of Patients With a History of Radiotherapy With Artificial Urinary Sphincters

open access: yesNeurourology and Urodynamics, EarlyView.
ABSTRACT Aims This study assessed how functional satisfaction and quality of life outcomes of the artificial urinary sphincter (AUS) were affected in patients who received radiation for prostate cancer. Methods A secondary analysis was conducted based on the Artificial Urinary Sphincter Clinical Outcomes (AUSCO) study, a prospective multi‐institutional
Vishu Chandrasekhar   +21 more
wiley   +1 more source

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