Results 171 to 180 of about 41,607 (263)

Global research dynamics in urea cycle disorders: a bibliometric study highlighting key players and future directions. [PDF]

Orphanet J Rare Dis
Wang Y, Wang X, Zhang H, Zhu B.
europepmc   +1 more source

Identification of rare variants causing urea cycle disorders: A clinical, genetic, and biophysical study. [PDF]

J Cell Mol Med, 2021
Liu F, Bao LS, Liang RJ, Zhao XY, Li Z, Du ZF, Lv SG.   +6 more
europepmc   +1 more source

Prescribing competence: The pros and cons of different methods for assessment

British Journal of Clinical Pharmacology, EarlyView.
Evaluating a medical graduate’s competence in rational prescribing is challenging. With the aim to guide and inspire teachers, this narrative review explores different methods that can be used to assess prescribing competence. Each method has its own advantages and disadvantages, and thus a mix of different assessment methods is needed throughout the ...
David J. Brinkman, Erik M. Donker, Jelle Tichelaar, on behalf of the CP4T programme   +3 more
wiley   +1 more source

Perspectives on long-term medical management of urea cycle disorders: insights from a survey of UK healthcare professionals. [PDF]

Orphanet J Rare Dis
Stepien KM, McSweeney M, Ochoa-Ferraro A, Vara R, Riley P, Smith M.   +5 more
europepmc   +1 more source

Decoding RNA regulation: Challenges and opportunities for RNA‐based therapies in Europe

British Journal of Clinical Pharmacology, EarlyView.
Abstract RNA‐based medicinal products represent a promising frontier in personalised medicine, offering sequence‐specific disease targeting at various molecular levels, yet their clinical translation in the European Union (EU) may be hindered by regulatory uncertainty around definitions and evidence requirements; this study therefore aims to identify ...
Olivia C. Lewis, Christine Leopold, Joyce M. Hoek, Raymond M. Schiffelers, Marie L. De Bruin   +4 more
wiley   +1 more source

Novel Treatment Strategy for Patients With Urea Cycle Disorders: Pharmacological Chaperones Enhance Enzyme Stability and Activity in Patient-Derived Liver Disease Models. [PDF]

J Inherit Metab Dis
Ramosaj A, Borsuk M, Underhaug J, Mathis D, Matsumoto S, Keogh A, Banz V, Pandey AV, Gougeard N, Rubio V, Martinez A, Allegri G, Poms M, Thöny B, Häberle J, Laemmle A.   +15 more
europepmc   +1 more source

Atomically precise metal cluster enzymes for pathological tissue regeneration

BMEMat, EarlyView.
Schematic illustration of atomically precise metal cluster enzymes (MCEs) for pathological tissue regeneration. Atomically precise MCEs can modulate biological processes, such as attenuation of inflammatory responses, eradication of bacterial pathogens, regulation of angiogenesis, and promotion of cell development.
Ziqiang Xiong, Zhifeng Shi, Mengqing Li, Jien Han, Haoyan Chen, Maofei Ran, Shaojian Zhong, Limin Ma, Chengyun Ning, Youzhun Fan, Zhenghua Tang, Peng Yu   +11 more
wiley   +1 more source

Mapping challenges in the accessibility of treatment products for urea cycle disorders: A survey of European healthcare professionals. [PDF]

J Inherit Metab Dis
Stolwijk NN, Häberle J, Huidekoper HH, Wagenmakers MAEM, Hollak CEM, Bosch AM, E‐IMD and MetabERN Working Group on the Real‐World Use of products for UCD Management.   +6 more
europepmc   +1 more source

Pt‐TiO2 mesoporous nanosystem co‐delivering phlorezin enables synergistic sonodynamic‐chemoimmunotherapy for hepatocellular carcinoma via directly targeting DDX5 and metabolic reprogramming modulation

BMEMat, EarlyView.
Abstract Hepatocellular carcinoma (HCC), ranking as the third leading cause of cancer‐related mortality globally, continues to pose significant therapeutic challenges. Here, we developed an innovative nanosystem, Phl@PT, based on Pt‐TiO2 nanoparticles for the co‐delivery of Phlorezin, presenting a novel approach for HCC treatment through sonodynamic ...
Kairui Liu, Bo Zhang, Yue Wan, Zhiru Yang, Abdusami Abdurahman, Mengxin He, Shengtao Hu, Yi Zhang, Xiaoke Li, Jin Chang, Jun Kang, Liren Liu, Wanghua Liu, Shao Li   +13 more
wiley   +1 more source

Understanding the Natural History and the Effects of Current Therapeutic Strategies on Urea Cycle Disorders: Insights from the UCD Spanish Registry. [PDF]

Nutrients
Martín-Hernández E, Bellusci M, Pérez-Mohand P, Correcher Medina P, Blasco-Alonso J, Morais-López A, de Las Heras J, Meavilla Olivas SM, Dougherty-de Miguel L, Couce ML, Villarroya EC, García Jiménez MC, Moreno-Lozano PJ, Vives I, Gil-Campos M, Stanescu S, Ceberio-Hualde L, Camprodón M, Cortès-Saladelafont E, López-Urdiales R, Murray Hurtado M, Márquez Armenteros AM, Sierra Córcoles C, Peña-Quintana L, Ruiz-Pons M, Alcalde C, Castellanos-Pinedo F, Dios E, Barrio-Carreras D, Martín-Cazaña M, García-Peris M, Andrade JD, García-Volpe C, de Los Santos M, García-Cazorla A, Del Toro M, Felipe-Rucián A, Comino Monroy MJ, Sánchez-Pintos P, Matas A, Gil Ortega D, Martín-Rivada Á, Bergua A, Belanger-Quintana A, Vitoria I, Yahyaoui R, Pérez B, Morales-Conejo M, Quijada-Fraile P.   +48 more
europepmc   +1 more source