Results 61 to 70 of about 6,607,545 (247)

Safety and Tolerability of Givinostat: Evidence From Real‐World and Clinical Practice

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective The aim of our study was to establish the prevalence of adverse events in a real‐world setting in boys living with Duchenne muscular dystrophy (DMD) treated with givinostat as part of an Expanded Access Program (EAP) in Italy. Methods The cohort included 90 ambulant boys, with age when treatment started between 6 and 23 years (mean ...
Marika Pane, Anna Capasso, Chiara Arpaia, Adele D’Amico, Emilio Albamonte, Federica Trucco, Maria Sframeli, Riccardo Masson, Francesca Magri, Luca Bello, Romina Venditti, Claudia Dosi, Michela Catteruccia, Michele Tosi, Claudio Bruno, Sonia Messina, Giacomo Comi, Elena Pegoraro, Valeria A. Sansone, Eugenio Mercuri   +19 more
wiley   +1 more source

The Initial Middle Eastern Experience With the Alterra Adaptive Pre-Stent: Single-Center Outcomes From Saudi Arabia

CJC Pediatric and Congenital Heart Disease
Background: The Alterra Adaptive Pre-Stent, used with the Edwards SAPIEN 3 valve, offers a novel transcatheter option for patients with severely dilated right ventricular outflow tracts (RVOTs) previously unsuitable for conventional transcatheter ...
Mohamed Al Nasef, FRCPCH, Bandar Al Shehri, MD, Khalid Al Naji, MD, Ahmed Al Zahrani, MD, Merna Atiyah, MD, Omar Abdulaziz Almohizy, MD, Saria Mansour Ahmed, MD, Khalid Al Najashi, MD   +7 more
doaj   +1 more source

AV nodal ablation in tricuspid atresia; anatomical challenges

Indian Pacing and Electrophysiology Journal, 2016
Patients with congenital heart disease often present unique challenges, especially in the electrophysiology laboratory. Here we present a case of a patient with medically refractory symptomatic atrial tachycardia, tricuspid atresia and a history of a ...
Jeffrey Munro, Krishnaswamy Chandrasekaran, Komandoor Srivathsan   +2 more
doaj   +1 more source

Early Clinical, Imaging, and Pathological Characteristics of SRPK3/TTN‐Digenic Myopathy

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective SRPK3/TTN‐digenic myopathy was recently established as a skeletal muscle myopathy caused by digenic inheritance. This study characterizes the early clinical presentation of SRPK3/TTN‐digenic myopathy in one previously reported and seven newly identified pediatric patients.
Rotem Orbach, Sandra Donkervoort, Carola Hedberg‐Oldfors, Giovanni Baranello, Dimah Saade, Precilla D'Souza, Ruchee Patel, Eva Michael, A. Reghan Foley, Diana Bharucha‐Goebel, S. Jin Haugland, Meghan McAnally, Omer Abdul Hamid, Katherine Chao, Ellen F. Macnamara, Alan H. Beggs, Anna Sarkozy, Juliane Mueller, Steven A. Moore, Richard S. Finkel, Cynthia J. Tifft, Francesco Muntoni, Anders Oldfors, Carsten G. Bönnemann   +23 more
wiley   +1 more source

Ventricular remodeling in ischemic heart failure stratifies responders to stem cell therapy

Stem Cells Translational Medicine, 2020
Response to stem cell therapy in heart failure is heterogeneous, warranting a better understanding of outcome predictors. This study assessed left ventricular volume, a surrogate of disease severity, on cell therapy benefit.
Satsuki Yamada, D. Kent Arrell, Christian S. Rosenow, Jozef Bartunek, Atta Behfar, Andre Terzic   +5 more
doaj   +1 more source

Spatial and Volumetric Characteristics of Glioblastoma: Associations With Clinical Presentation and Survival

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective We aim to comprehensively analyze how regional tumor and edema characteristics are associated with clinical presentations and survival outcomes in a large cohort of glioblastoma patients. Methods Patients with IDH‐wildtype glioblastoma who received brain MRI from 2010 to 2023 were included.
Daniel J. Zhou, Marc Jaskir, Julian Gal, Yombe Fonkeu, Christos Davatzikos, MacLean P. Nasrallah, Jose Garcia, Rubo Xing, Stephen J. Bagley, Arati Desai, Richard E. Phillips, Steven Tobochnik, Colin A. Ellis, James J. Gugger, Nishant Sinha, Joel M. Stein, Kathryn A. Davis   +16 more
wiley   +1 more source

Use of the Archimedes navigation system to diagnose peripheral pulmonary lesions: preliminary Italian results

Frontiers in Oncology
Diagnosis of peripheral pulmonary lesions (PPL) is one of the most challenging fields in early lung cancer diagnosis. Despite novel techniques and new approaches to the periphery of the lung, almost 25% of PPL remains undiagnosed.
Filippo Lanfranchi, Laura Mancino, Gabriele Foltran, Lorenzo Nicole’, Lucio Michieletto   +4 more
doaj   +1 more source

Plasma EV Proteomics Identifies ECM Remodeling and Inflammatory Proteins LUM and C7 as Candidate Biomarkers in FSHD

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Facioscapulohumeral muscular dystrophy (FSHD) is one of the most debilitating and common muscular dystrophies. Despite its severity, no approved therapy exists for FSHD patients. However, several therapeutic candidates are currently under development, and some have recently entered clinical trials, marking the need for reliable ...
Mustafa Bilal Bayazit, Chiranth K. Nagaraj, Jackson S. Newell, Kim Truc Nguyen, Xilal Y. Rima, Jacob Doon‐Ralls, Eduardo Reátegui, Jeffrey M. Statland, Rabi Tawil, Kevin M. Flanigan, Scott Q. Harper, Nizar Y. Saad   +11 more
wiley   +1 more source

Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective There are limited real‐world data regarding the safety and effectiveness of onasemnogene abeparvovec (OA; Zolgensma) infusion, a one‐time gene replacement therapy, for Japanese patients with spinal muscular atrophy (SMA). We aimed to improve understanding of the real‐world outcomes for OA in Japan.
Kayoko Saito, Kamal Benguerba, Ken Tsuchida, Kazushige Yazawa, Isao Tsumiyama, Hiromitsu Kayama, Sandra P. Reyna, Farid Khan, Richard S. Finkel   +8 more
wiley   +1 more source

Better cardiac care measures for Aboriginal and Torres Strait Islander people: first national report 2015 [PDF]

, 2015
Mortality from cardiac conditions for Indigenous Australians decreased by 41% between 1998 and 2012, while access to cardiac care has improved over time.
Australian Institute of Health and Welfare
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