Results 161 to 170 of about 161,608 (297)

Five‐Year Outcomes With Delandistrogene Moxeparvovec in Patients With Duchenne Muscular Dystrophy: A Phase 1/2a Study

Muscle &Nerve, EarlyView.
ABSTRACT Aims We report 5‐year results from a phase 1/2a study of delandistrogene moxeparvovec, a recombinant adeno‐associated virus serotype rh74 vector‐based gene therapy for Duchenne muscular dystrophy (DMD), with post hoc analyses contextualizing functional outcomes. Methods Four ambulatory patients with DMD (≥ 4–< 8 years at enrollment) entered an
Jerry R. Mendell, Zarife Sahenk, Linda P. Lowes, Megan A. Iammarino, Lindsay N. Alfano, Craig M. McDonald, James E. Signorovitch, Jim Jin, Jing Li, Stefanie Mason, Louise R. Rodino‐Klapac   +10 more
wiley   +1 more source

A Novel NPHS1-Associated Phenotype Characterized by Recurrent Transient Proteinuria. [PDF]

Case Rep Nephrol
Tanaka E, Konomoto T, Sakaguchi H, Kurogi J, Sakakibara N, Nozu K, Moritake H.   +6 more
europepmc   +1 more source

IgG Subclass (IgG1‐4) and IgA Autoantibody Profiles Against Muscle‐Specific Kinase in a Greek Cohort

Muscle &Nerve, EarlyView.
ABSTRACT Introduction/Aims Muscle‐specific kinase myasthenia gravis (MuSK‐MG) is an autoimmune neuromuscular disorder predominantly mediated by IgG4 autoantibodies disrupting MuSK signaling. The contribution of other isotypes remains incompletely defined. We characterized the serological profile of a Greek cohort of MuSK‐MG patients.
Sofia‐Natsοuko Gkotzamani, Maria Pechlivanidou, Francisca Faber, Cansu Elmas, Vasiliki Zouvelou, Elisabeth Chroni, Dimitrios Tzanetakos, Maria Kourouvani, Marlene Wolfsgruber, Ioannis Zaganas, Dimitra Kolovou, Michael Rentzos, Ariadne Daponte, Maria Chondrogiorgi, Spyridon Konitsiotis, Georgios Nicolaou, Stavroula Salakou, Sotirios Giannopoulos, Konstandinos Lazaridis, Georgios Tsivgoulis, Socrates Tzartos, Inga Koneczny, John S. Tzartos   +22 more
wiley   +1 more source

Kidney transplantation in sub-Sahara African countries: challenges and lessons from the experience of Cameroon: a preliminary report of the first 17 pioneer cases. [PDF]

Ren Fail
Djientcheu VP, Ashuntantang G, Nono A, Kamga J, Mba S, Ngongang MP, Tebere H, Ngoundjou A, Aghogue T, Essissima F, Tiam M, Fokou M.   +11 more
europepmc   +1 more source

Type I interferon overactivation in sera of patients with anti‐MDA5‐positive dermatomyositis‐associated interstitial lung disease

Rheumatology &Autoimmunity, EarlyView.
Sera from patients with anti‐MDA5‐positive dermatomyositis‐associated interstitial lung disease (ILD) exhibit significantly higher type I interferon (IFN‐I) bioactivity and interferon‐stimulated gene‐inducing activity compared to anti‐ARS‐positive ILD and healthy controls.
Shohei Nakamura, Yasuhiro Katsumata, Yuko Okamoto, Masayoshi Harigai   +3 more
wiley   +1 more source

Cold agglutinin syndrome secondary to Epstein-Barr virus reactivation during corticosteroid therapy for minimal change nephrotic syndrome in a patient with human immunodeficiency virus infection. [PDF]

CEN Case Rep
Bamba H, Katagiri D, Shimada K, Otani H, Eriguchi N, Katayama Y, Koizumi M, Suzuki M, Takano H.   +8 more
europepmc   +1 more source

Belgian bovine veterinarians' practices and opinions on udder health‐related antimicrobial use and services

Veterinary Record, EarlyView.
Abstract Background Changing legislation regarding antimicrobial consumption (AMC) forces Flemish bovine veterinary practitioners (BVP) to rethink their business models and shift their on‐farm practices from curative to preventive. However, little is known about their current practices and opinions regarding antibiotic stewardship, mastitis treatment ...
Lien Creytens, Sofie Piepers, Sarne De Vliegher   +2 more
wiley   +1 more source

Drug Transport in a Liquid-Crystalline Supramolecular Hydrogel: Diffusion Mechanisms Revealed by PGSE NMR. [PDF]

Pharmaceutics
Wang W.
europepmc   +1 more source

Treatment of discospondylitis in dogs: A systematic review

Veterinary Record, EarlyView.
Abstract Background Both medical and surgical techniques have been reported to manage canine discospondylitis, although data on clinical effectiveness and long‐term prognosis remain limited. Methods A systematic review of studies on treatment approaches, follow‐up data and investigation methods was conducted.
Vasileios Ioannis Vallios, Evdokia Sourla, Daniel Low, Holger A. Volk, Marios Charalambous   +4 more
wiley   +1 more source

Reversibility of calcinosis in anti-NXP2-positive refractory dermatomyositis treated with TNF-α blockade: a brief report. [PDF]

Front Med (Lausanne)
Huang H, Yu H, Fan Z, Guo Y, Ma H, Huang N, Ma L.   +6 more
europepmc   +1 more source