Results 71 to 80 of about 88,754 (289)

Engineered GM1 Intersects Between Mitochondrial and Synaptic Pathways to Ameliorate ALS Pathology

Advanced Science, EarlyView.
Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disease driven by genetic and molecular disruptions affecting energy balance, protein homeostasis, and stress responses in nerve cells. Studies using human and rodent models identified convergent defects in mitochondria and synaptic function.
Federica Pilotto, Tristan Dellazizzo Toth, Silvano Bond, Alexander Schmitz, Rim Diab, Sara Y. Ngo Tenlep, Brian Mooney, Silvia Erni, Martina Schobesberger, Olivier Scheidegger, Camille Peitsch, Smita Saxena   +11 more
wiley   +1 more source

Access to Life-Saving Medicines and Intellectual Property Rights: An Ethical Assessment [PDF]

, 2011
© 2011 Cambridge University Press. Online edition of the journal is available at http://journals.cambridge.org/action/displayJournal?jid=CQHDying before one’s time has been a prominent theme in classic literature and poetry.
Aquinas, Aquinas, Aquinas, Brown, DORIS SCHROEDER, Finnis, Jentzsch, Leisinger, Locke, Mamunes, May, Nozick, Pang, PETER SINGER, Pogge, Pogge, Rawls, Rifkin, Schroeder, Selgelid, Shelley   +20 more
core   +2 more sources

International experience in drug assessment for rare diseases: flexibility of approaches

Реальная клиническая практика: данные и доказательства
Relevance. The regulation of orphan drugs plays a key role in the development of medicines for rare diseases that affect only a small percentage of the population. This specialized regulatory framework is designed to encourage pharmaceutical companies to
K. S. Radaeva, A. S. Kolbin
doaj   +1 more source

Common drug review recommendations for orphan drugs in Canada: basis of recommendations and comparison with similar reviews in Quebec, Australia, Scotland and New Zealand

Orphanet Journal of Rare Diseases, 2018
Background Public payer reimbursement for non-oncology drugs in Canada, including orphan drugs, is based on recommendations by the Common Drug Review (CDR) (with the exception of Quebec).
John I. McCormick, L. Diana Berescu, Nabil Tadros   +2 more
doaj   +1 more source

Universal Antibody‐Engineered Lipid Nanoparticles Potentiate Chemo‐Immunotherapy Against Triple‐Negative Breast Cancer by Reprogramming Tumor Cell Metabolism

Advanced Science, EarlyView.
A universal ROR1 antibody‐engineered lipid nanoparticle (LNP) is constructed by sequential drug encapsulation and antibody conjugation. The LNP not only enables enhanced chemotherapeutic efficiency by targeting drug delivery and precise localization of deep‐seated tumors through NIR‐II fluorescence imaging, but also remodels immunosuppressive TME ...
Yeneng Dai, Jiaqi Wang, Yu Liu, Guanda Jiao, Yuheng Gu, Yang Liu, Shengyu Fu, Xing Fan, Jialin Li, Ziang Guo, Kam Tong Leung, Lipeng Zhu, Qi Zhao   +12 more
wiley   +1 more source

Regulating Rare Disease: Safely Facilitating Access to Orphan Drugs [PDF]

, 2018
While approximately one in ten Americans suffers from a rare disease, only 5 percent of rare diseases have a U.S. Food and Drug Administration (FDA) approved treatment.
Bannister, Julien B.
core   +1 more source

No difference in between-country variability in use of newly approved orphan and non- orphan medicinal products - a pilot study

Orphanet Journal of Rare Diseases, 2009
Background Regulators and payers have to strike a balance between the needs of the patient and the optimal allocation of resources. Drugs indicated for rare diseases (orphan medicines) are a special group in this context because of their often high per ...
Bloechl-Daum Brigitte, Leufkens Hubert GM, Heemstra Harald E, Stolk Pieter, Heerdink Eibert R   +4 more
doaj   +1 more source

Microglial GPR35 Ameliorates Epileptogenesis and Neuroinflammation via PDGFA Domain 2 Signaling

Advanced Science, EarlyView.
Activation of microglial G protein–coupled receptor 35 (GPR35) by L‐kynurenic acid (L‐Kyna) initiates a platelet‐derived growth factor A (PDGFA)–dependent phosphoinositide 3‐kinase–protein kinase B (PI3K–AKT) signaling cascade that dampens hippocampal neuroinflammation, thereby restraining epileptogenesis, lowering seizure susceptibility, and ...
Qi Wang, Tingting Qu, Qibing Sun, Ran Li, Junfei Dong, Yuming Du, Ziyin Xuan, Lei Wang, Hanli Li, Jianyun Sun, Fangliang Chen, Jinshuai Liu, Zifan Yang, Jianxiang Lei, Qian Yang, Bin Wang, Zhiming Zhou, Yu Wang   +17 more
wiley   +1 more source

T Cell Exhaustion in Cancer Immunotherapy: Heterogeneity, Mechanisms, and Therapeutic Opportunities

Advanced Science, EarlyView.
T cell exhaustion limits immunotherapy efficacy. This article delineates its progression from stem‐like to terminally exhausted states, governed by persistent antigen, transcription factors, epigenetics, and metabolism. It maps the exhaustion landscape in the TME and proposes integrated reversal strategies, providing a translational roadmap to overcome
Yang Yu, Xiaoyu Yao, Qingyang Wang, Mengrui Yang, Runze Li, Jiangjiang Qin, Jing Zhuang, Changgang Sun   +7 more
wiley   +1 more source

Understanding Disease Heterogeneity and Patient Characteristics in Patients with Amyotrophic Lateral Sclerosis (ALS) [PDF]

, 2019
Background: Amytrophic lateral sclerosis (ALS) is a fatal neurologic disease that is projected to double in worldwide incidence in the next 20 years.
Mathew, Veena
core