Results 131 to 140 of about 358,140 (209)

Phase 3 Randomized Trial Results of DTX401 AAV Gene Therapy for the Treatment of GSDIa

open access: yesJournal of Inherited Metabolic Disease, Volume 49, Issue 5, September 2026.
ABSTRACT Glycogen storage disease type Ia (GSDIa) is a rare, life‐threatening inherited carbohydrate metabolism disorder caused by biallelic pathogenic G6PC gene variants resulting in deficiency of glucose‐6‐phosphatase. DTX401 is an investigational AAV8 vector containing the human G6PC gene. DTX401‐CL301 is a pivotal, phase 3, double‐blind, randomized,
John J. Mitchell   +37 more
wiley   +1 more source

Antitumor Activity of Combined Topoisomerase Inhibition and FLASH Radiotherapy in Head and Neck Carcinoma Biomodels

open access: yesMedComm, Volume 7, Issue 9, September 2026.
We report a proof‐of‐concept combination of topoisomerase inhibition and FLASH radiotherapy showing enhanced antitumor activity in head and neck carcinoma biomodels, supporting further investigation of this strategy in more clinically representative models and treatment settings. ABSTRACT Locally advanced head and neck carcinoma remains associated with
Patrizia Sarogni   +17 more
wiley   +1 more source

The Hidden Burden of Hemifacial Spasm: A Systematic Review of Non‐Motor Symptoms

open access: yesMovement Disorders Clinical Practice, Volume 13, Issue 9, Page 2059-2071, September 2026.
Abstract Background Hemifacial spasm (HFS) is a chronic neurological disorder characterized by involuntary contractions of facial muscles. Traditionally regarded as a motor condition, HFS encompasses a spectrum of non‐motor symptoms that are often overlooked but significantly affect patients’ quality of life.
Miriam Carvalho Soares   +2 more
wiley   +1 more source

Beyond Humor: Laughter as a Cardiovascular Stimulus. [PDF]

open access: yesArq Bras Cardiol
Stein R, Andrade CDS, Ferrari F.
europepmc   +1 more source

Fibrosis pulmonar intersticial centrada en las vías aéreas asociada a inhalación de productos de limpieza

open access: yes, 2006
La fibrosis pulmonar intersticial centrada en las vías aéreas es una entidad clinicopatológica poco conocida y de reciente descripción, que se caracteriza anatomopatológicamente por fibrosis bronquiolar y fibrosis pulmonar intersticial localizada ...
Serrano, Mario
core  

FXTAS and the Spectrum of FMR1 Premutation‐Associated Phenotypes in Latin America: A Scoping Review

open access: yesMovement Disorders Clinical Practice, Volume 13, Issue 9, Page 2045-2058, September 2026.
Abstract Background Fragile X–associated tremor/ataxia syndrome (FXTAS) is a late‐onset neurodegenerative disorder caused by FMR1 premutation expansions (55–200 CGG repeats). Although well described in populations of predominantly European ancestry, FXTAS remains poorly characterized in Latin America due to limited awareness, restricted access to ...
Amy Schmidmajer   +6 more
wiley   +1 more source

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