Results 31 to 40 of about 22,199 (155)

Pyruvate Dehydrogenase Kinase as a Novel Therapeutic Target in Oncology

Frontiers in Oncology, 2013
Current drug development in oncology is non-selective as it typically focuses on pathways essential for the survival of all dividing cells. The unique metabolic profile of cancer, which is characterized by increased glycolysis and suppressed ...
Gopinath eSutendra, Evangelos D. Michelakis   +1 more
doaj   +1 more source

Revolutionizing Drug Delivery: The Impact of Advanced Materials Science and Technology on Precision Medicine

Pharmaceutics
Recent progress in material science has led to the development of new drug delivery systems that go beyond the conventional approaches and offer greater accuracy and convenience in the application of therapeutic agents.
Mohamed El-Tanani, Shakta Mani Satyam, Syed Arman Rabbani, Yahia El-Tanani, Alaa A. A. Aljabali, Ibrahim Al Faouri, Abdul Rehman   +6 more
doaj   +1 more source

The work, goals, challenges, achievements, and recommendations of orphan medicinal product organizations in India: an interview-based study

Orphanet Journal of Rare Diseases, 2019
Background Orphan medicinal products (OMPs) are intended for the diagnosis, prevention, management or treatment of rare diseases (RDs). Each RD affects only a small fraction of the population, and therefore, historically, industry hesitated to undertake ...
Mohua Chakraborty Choudhury, Gayatri Saberwal   +1 more
doaj   +1 more source

Venture philanthropy in rare disease therapy

Rare
Purpose: Rare diseases affect ∼350 million people globally, yet 94 % of those affected lack effective treatments due to funding gaps and high R&D costs.
JA Levine
doaj   +1 more source

The outcome of more than 3 decades of orphan drug development in alpha-1-antitypsin deficiency – a cross sectional analysis of the FDA Orphan Drug Product designation database

, 2022
Abstract Introduction Alpha-1 antitrypsin deficiency (AATD, OMIM #613490) is a rare metabolic disorder affecting lungs and liver. The purpose of this study is to assess the impact of the US orphan drug act on AATD by providing a quantitative clinical-regulatory insight into the status of FDA orphan drug approvals and designations for compounds ...
Franziska C Trudzinski, Maria Ada Presotto, Emanuel Buck, Felix J. Herth, Markus Ries   +4 more
openaire   +1 more source

New Approaches and Strategies for the Repurposing of Iron Chelating/Antioxidant Drugs for Diseases of Free Radical Pathology in Medicine

Antioxidants
There is an urgent need for new approaches and strategies for the introduction of antioxidant drugs in medicine. Despite hundreds of clinical trials with potential antioxidants, no antioxidant drugs have so far been developed for clinical use; this is ...
George J. Kontoghiorghes
doaj   +1 more source

Failures to further developing orphan medicinal products after designation granted in Europe: an analysis of marketing authorisation failures and abandoned drugs [PDF]

BMJ Open, 2017
ObjectivesThe research and development process in the field of rare diseases is characterised by many well-known difficulties, and a large percentage of orphan medicinal products do not reach the marketing approval.This work aims at identifying orphan medicinal products that failed the developmental process and investigating reasons for and possible ...
Giannuzzi, Viviana, Landi, Annalisa, Bosone, Enrico, Giannuzzi, Floriana, Nicotri, Stefano, Torrent-Farnell, Josep, Bonifazi, Fedele, Felisi, Mariagrazia, Bonifazi, Donato, Ceci, Adriana   +9 more
openaire   +2 more sources

New Insights in Cushing Disease Treatment With Focus on a Derivative of Vitamin A

Frontiers in Endocrinology, 2018
Cushing’s disease (CD) is an endocrine disorder originated by a corticotroph tumor. It is linked with high mortality and morbidity due to chronic hypercortisolism. Treatment goals are to control cortisol excess and achieve long-term remission, therefore,
Mariana Fuertes, Julieta Tkatch, Josefina Rosmino, Leandro Nieto, Mirtha Adriana Guitelman, Eduardo Arzt, Eduardo Arzt   +6 more
doaj   +1 more source

Building local research and development capacity for the prevention and cure of neglected diseases: the case of India

Bulletin of the World Health Organization, 2001
This paper examines the proposal to build research and development (R & D) capabilities for dealing with neglected infectious and tropical diseases in countries where they are endemic, as a potentially cost- and time- effective way to fill the gap ...
Kettler Hannah E., Modi Rajiv
doaj  

Rhodopsin orphan GPCR20 interacts with neuropeptides and directs growth, sexual differentiation, and egg production in female Schistosoma mansoni

Microbiology Spectrum
Schistosomes are parasitic flatworms that cause schistosomiasis, a neglected tropical disease of worldwide importance. Since standard treatment of schistosomiasis relies on a single drug, praziquantel, alternative drugs are needed.
Xuesong Li, Oliver Weth, Martin Haimann, Max F. Möscheid, Theresa S. Huber, Christoph G. Grevelding   +5 more
doaj   +1 more source