Results 241 to 250 of about 324,761 (353)

[Translated article] Use of a Spinal Cord Stimulator to Treat Livedoid Vasculopathy: Effective Control of an Untreatable Disease

Leonor Revelles‐Peñas, S. Pastor-Navarro, AA. López-Piñero, Virgínia Velasco-Tirado   +3 more
openalex   +1 more source

Cost‐utility analysis of nusinersen–risdiplam switch in patients with spinal muscular atrophy in Croatia: A discrete event simulation model

British Journal of Clinical Pharmacology, EarlyView.
Introduction In recent years, the treatment of spinal muscular atrophy (SMA), a rare disease, has significantly progressed, improving patients' survival and overall quality of life. However, current SMA treatments are expensive, and some (nusinersen) are very inconvenient for patients.
Andrej Belančić, Ivana Stević, Elvira Meni Maria Gkrinia, Dinko Vitezić, Slobodan Janković   +4 more
wiley   +1 more source

Diagnostic and therapeutic challenges of isolated spinal vasculitis: A case report. [PDF]

Exp Ther Med
Yao Y, Dong GH, Tian DC.
europepmc   +1 more source

The evolving therapeutic landscape of spinal muscular atrophy – A scoping review of investigational agents, emerging delivery technologies and strategic innovations

British Journal of Clinical Pharmacology, EarlyView.
Spinal muscular atrophy (SMA) is a severe neuromuscular disease with emerging therapeutic complexity. This review aims to systematically map the global pipeline of investigational treatments for SMA. Using ClinicalTrials.gov and complementary international registries, we identified 21 planned or ongoing interventional trials from 2020 to 2025 targeting
Andrej Belančić, Patrick Eustaquio, Elvira Meni Maria Gkrinia, Ivana Stević, Eugen Javor, Yun Wah Lam, Slobodan Janković, Dinko Vitezić   +7 more
wiley   +1 more source

Comparison of AAV9-driven motor neuron transduction following different CNS-directed delivery methods in mice. [PDF]

Sci Rep
Mortimer AJ, Sander CF, Parmar AR, Williams AJ, Azzouz M, Hautbergue GM, Shaw PJ, Ferraiuolo L, Mead RJ.   +8 more
europepmc   +1 more source

Mapping opioid exposure through prescription data and postmortem analysis of opioid drugs in multiple tissues

British Journal of Clinical Pharmacology, EarlyView.
Background and Purpose Although opioids are central to end of life (EoL) care, tissue‐level opioid exposure remains poorly understood. The objective of this study was to characterize the relationship between prescription‐derived morphine equivalent daily dose (MEDD) and measured morphine concentrations across multiple organs.
Niamh Higgins, Alan Wells, Nimish Patel, Nicole Muttera, Mattia Trunfio, Alessandra Manca, Amedeo De Nicolò, Micol Ferrara, Stefano Bonora, Jessica Cusato, Alice Palermiti, Antonio D'Avolio, Marissa Borochoff, Patricia K. Riggs, Elizabeth Hastie, Rabia S. Atayee, Stephanie Solso, Cheryl Dullano, Vanessa Gomez‐Moreno, Tara Tenenbaum, Robert Deiss, Davey M. Smith, Antoine Chaillon, Sara Gianella   +23 more
wiley   +1 more source

Impaired dynein function preserves spinal interneuron survival and positioning in an ALS-like mouse model. [PDF]

PLoS One
Christoforidou E, Rowe JS, Simoes FA, Cassel R, Dupuis L, Leigh PN, Hafezparast M.   +6 more
europepmc   +1 more source

Active surveillance of drug safety in healthcare data: Sequential monitoring of bacterial and serious urinary tract infection risk in sodium‐glucose cotransporter 2 inhibitor users

British Journal of Clinical Pharmacology, EarlyView.
Abstract Introduction Active surveillance of adverse events using healthcare data is emerging as complementary to the monitoring of spontaneous reports and stand‐alone pharmacoepidemiologic studies. The risk of urinary tract infections (UTIs) was listed as a special warning for sodium‐glucose cotransporter‐2 inhibitors (SGLT2is) when marketed in Europe
Haoxin Le, Dilsad Simay Peker, Lukas Frederic Westphal, Nikolai Brun, Christine Erikstrup Hallgreen, Morten Andersen   +5 more
wiley   +1 more source

Longitudinal extensive anti-AMPA-GluR3-related transverse myelitis and favorable therapeutic response: A case report and review of literature. [PDF]

World J Clin Pediatr
Cucuzza ME, Pavone P, Garozzo MT, Finocchiaro MC, Greco F, Smilari P, Di Nora A, Ruggieri M, Di Stefano VA.   +8 more
europepmc   +1 more source

Adaptive Machine Learning Framework for Optimizing the Affinity Purification of Adeno‐Associated Viral Vectors

Biotechnology and Bioengineering, EarlyView.
ABSTRACT Adeno‐associated viral (AAV) vectors for gene therapy are becoming integral to modern medicine, providing therapeutic options for diseases once deemed incurable. Currently, viral vector purification is a critical bottleneck in the gene therapy industry, impacting product efficacy and safety as well as accessibility and cost to patients ...
Kelvin P. Idanwekhai, Shriarjun Shastry, Morgan R. Hurst, Arianna Minzoni, Eduardo Barbieri, Luke Remmler, Eugene N. Muratov, Michael A. Daniele, Stefano Menegatti, Alexander Tropsha   +9 more
wiley   +1 more source