Results 151 to 160 of about 6,815,015 (311)

Onasemnogene Abeparvovec in Type I Spinal Muscular Atrophy: 24‐Month Follow‐Up From the Italian Registry

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Onasemnogene abeparvovec (OA) is an AAV9‐based gene therapy for spinal muscular atrophy type I (SMA I). Real‐world outcomes show increased response variability compared to clinical trials, and follow‐up data beyond 12–18 months are limited.
Marika Pane, Giorgia Coratti, Chiara Cutrì, Antonio Varone, Riccardo Masson, Adele D'Amico, Valeria Sansone, Sonia Messina, Federica Ricci, Chiara Ticci, Claudio Bruno, Caterina Agosto, Francesca Benedetti, Antonella Pini, Sabrina Siliquini, Massimiliano Filosto, Alberto Zambon, Ilaria Bitetti, Maria Rosaria Manna, Claudia Dosi, Riccardo Zanin, Stefano Parravicini, Roberto De Sanctis, Giulia Stanca, Michela Catteruccia, Michele Tosi, Irene Mizzoni, Emilio Albamonte, Valentina Franchino, Maria Sframeli, Ilaria Cavallina, Elena Procopio, Michele Sacchini, Simone Morando, Noemi Brolatti, Federica Trucco, Gaia Scarpini, Elena Briganti, Beatrice Berti, Concetta Palermo, Daniela Leone, Stefano C. Previtali, Eugenio Mercuri, the ITASMAC working group   +43 more
wiley   +1 more source

Study on nutritional status and incidence of sarcopenia in elderly patients with chronic heart failure. [PDF]

Asia Pac J Clin Nutr
Song S, Du H, Lu X, Luo B, Li Z.
europepmc   +1 more source

A Depolarizing Leak in Sodium Bicarbonate Cotransporter NBCe1 Causes Brain Edema

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objectives SLC4A4 encodes electrogenic sodium bicarbonate cotransporter NBCe1, prominently expressed in kidney and brain. Recessive loss‐of‐function variants in SLC4A4 cause proximal renal tubular acidosis, no brain edema. In the brain, NBCe1 is expressed by astrocytes, where it regulates pH and mediates astrocyte volume changes.
Quinty Bisseling, Mark D. Parker, Sven Kerst, Richard A. Pasternack, Jacob Tondreau, Marjolein Breur, Gemma M. van Rooijen‐van Leeuwen, Davide Tonduti, Ettore Salsano, Alejandra Darling, Joanna A. E. van Wijk, Susanna Törnroth‐Horsefield, Marianna Bugiani, Petra J. W. Pouwels, Quinten Waisfisz, Marjo S. van der Knaap, Rogier Min   +16 more
wiley   +1 more source

Predictors of carotenoid status in New Zealand children using carotenoid reflection score: a cross-sectional study. [PDF]

Asia Pac J Clin Nutr
Rapson J, Rush E, McLean R, Fuavao K, Wall C, Eyles H.   +5 more
europepmc   +1 more source

Exploring Patients’ Profiles Associated With the Resolution of Acute Calcium Pyrophosphate Arthritis Treated With Colchicine and Prednisone: Post Hoc Analysis of a Randomized Controlled Trial

Arthritis Care &Research, EarlyView.
Objective The objective was to identify factors determining acute arthritis resolution and safety with colchicine and prednisone in acute calcium pyrophosphate (CPP) crystal arthritis. Methods We conducted a post hoc analysis of the COLCHICORT trial, which compared colchicine and prednisone for the treatment of acute CPP crystal arthritis, using a ...
Tristan Pascart, Laurène Norberciak, Pascal Richette, Pierre Robinet, Aurore Pacaud, Gauthier Marchasson, Thibault Rabin, Hélène Luraschi, Pierre Maciejasz, Anne‐France Georgel, Augustin Latourte, Hang‐Korng Ea, Sébastien Ottaviani, Charlotte Jauffret, Vincent Ducoulombier   +14 more
wiley   +1 more source

Personalized Surgical Tactics for an Adult Patient with Mitral Insufficiency and Dextrocardia with Situs Inversus Totalis. [PDF]

Braz J Cardiovasc Surg
Kozlov BN, Petlin KA, Lelik EV, Afanasieva NL, Arsenyeva YA, Chernykh YN, Kim EB.   +6 more
europepmc   +1 more source

Clinical Outcomes of Patients Within the Rheumatoid Arthritis Care Pathway Cohort at a Tertiary Care Integrated Delivery Network: A Comparison to Usual Care

Arthritis Care &Research, EarlyView.
Objective We aimed to compare clinical outcomes between patients in the Allegheny Health Network rheumatoid arthritis (RA) care pathway and patients receiving usual care. Methods The care pathway initiative implements guideline‐based best practice alongside multidisciplinary team‐based care.
Tarun Sharma, Tyson S. Barrett, Teigan Dwyer, Nancy Campbell, Jessica Heintzinger, Ellen Kraemer, Adam Dore, Susan Manzi   +7 more
wiley   +1 more source

Downregulation of Organ-Derived Activin A Attenuates Muscle Atrophy and Intramuscular Fat Infiltration in Cancer Cachexia Mice. [PDF]

J Cachexia Sarcopenia Muscle
Wang C, Gao L, Xue R, Su J, Li H, Yang W, Tang Y, Su Z, Min S, Tang C, Zhu Y, Mu B, Speakman JR, Xie X, Li Z.   +14 more
europepmc   +1 more source

Financial Distress and Its Determinants in Rheumatoid Arthritis

Arthritis Care &Research, EarlyView.
Objective To quantify the degree of financial distress and identify its determinants in adults with rheumatoid arthritis (RA) given the frequent prolonged use of expensive disease‐modifying therapies. Methods We identified adults enrolled in the FORWARD databank with either RA or noninflammatory musculoskeletal disease (NIMSKD) completing the ...
Amber Brown Keebler, Yunju Im, Sofia Pedro, Ted R. Mikuls, Edward S. Peters, Kaleb Michaud   +5 more
wiley   +1 more source

Heart Failure Risk in HIV: From Immunodeficiency to Metabolic Complexity. [PDF]

Circ Heart Fail
Shakil SS, Hsue PY.
europepmc   +1 more source