Results 141 to 150 of about 1,419,402 (298)

orphan drug

open access: yes
Citation: 'orphan drug' in the IUPAC Compendium of Chemical Terminology, 5th ed.; International Union of Pure and Applied Chemistry; 2025. Online version 5.0.0, 2025. 10.1351/goldbook.12699 • License: The IUPAC Gold Book is licensed under Creative Commons Attribution-ShareAlike CC BY-SA 4.0 International for individual terms.
openaire   +3 more sources

Artificial Intelligence‐Driven Natural Product Drug Discovery: From Computational Genome Mining to Clinical Translation

open access: yesMedicinal Research Reviews, EarlyView.
ABSTRACT Natural products (NPs) have historically yielded numerous therapeutic agents, yet their integration into modern drug discovery has been constrained by chemical complexity, low abundance, laborious dereplication, and limited target annotation.
Antonio Lavecchia
wiley   +1 more source

Ineffectiveness of immune checkpoint inhibitors in prostate cancer: is it just a question of ‘‐mabs’?

open access: yesThe Journal of Pathology, EarlyView.
Abstract Prostate cancer (PCa) is the second most common type of neoplasm, currently treated with a variety of therapeutic agents. Although advances in treatment over the past decade have improved overall survival, patients with advanced PCa eventually progress from hormone‐sensitive PCa to metastatic castration‐resistant PCa.
Giuseppe Schepisi   +8 more
wiley   +1 more source

Orphan Drug Development: Incentives Under the Orphan Drug Act [PDF]

open access: yes, 2015
After describing the intellectual property and regulatory environment for orphan drugs in the United States, this thesis compares the investment decisions in the orphan drug market with the larger pharmaceutical industry.
Smith, Sara H D
core   +1 more source

Gene turnover in the common ancestor of all C4 grasses

open access: yesPLANTS, PEOPLE, PLANET, EarlyView.
Understanding how plants evolve more efficient photosynthesis is important in a warming world where improving crop productivity and resilience is a global priority. By generating the first reference genomes for an early‐diverging group of grasses called the Aristidoideae, we were able to reconstruct the genetic makeup of the last common ancestor of all
Lara Pereira   +6 more
wiley   +1 more source

Plant responses to iron nutrition with emphasis on the underutilized crop tef (Eragrostis tef): A review

open access: yesPLANTS, PEOPLE, PLANET, EarlyView.
Iron (Fe) deficiency limits the physical health, cognitive development, and economic output of billions of people globally, especially children and productive‐age women. Tef is an underutilized and stress‐resilient crop, which is becoming popular for its grains containing high bioavailable Fe and has the potential to mitigate Fe deficiency.
Muhammad Numan   +2 more
wiley   +1 more source

Arzneimittel für seltene Krankheiten (orphan drugs) : Das schweizerische Heilmittelrecht im Vergleich mit der orphan drug Regulierung der EU [PDF]

open access: yes, 2012
Arzneimittel für seltene Krankheiten (orphan drugs) lassen sich unter normalen Marktbedingungen kaum kostendeckend erforschen, entwickeln und vermarkten, was zu einem Mangel an geeigneten Therapien und zugelassenen Arzneimitteln für seltene ...
Sprecher, Franziska
core  

Granzyme K: A new frontier in systemic lupus erythematosus

open access: yesRheumatology &Autoimmunity, EarlyView.
The role of Granzyme K in the pathogenesis of systemic lupus erythematosus and its clinical translational significance. Abstract Systemic lupus erythematosus (SLE) is a complex autoimmune disorder driven by dysregulated adaptive and innate immunity and aberrant complement activity.
Zhongyu Chen   +5 more
wiley   +1 more source

Artificial intelligence empowers targeted protein degradation: Core technological innovations, multi‐scenario applications, and translational prospects

open access: yesSmart Molecules, EarlyView.
AI is transforming TPD by improving the design, prediction, and optimization of degraders such as PROTACs, molecular glues, and LYTACs. This review summarizes key AI‐driven advances, highlights applications across drug discovery stages, and discusses remaining challenges and future directions for accelerating the development of therapies against ...
Shuanglin Qin   +10 more
wiley   +1 more source

Adenylosuccinic Acid: An Orphan Drug with Untapped Potential. [PDF]

open access: yesPharmaceuticals (Basel), 2023
Rybalka E   +5 more
europepmc   +1 more source

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