Results 51 to 60 of about 22,884 (253)

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

Paediatric development of radiopharmaceutical imaging agents and radioligand therapeutics

British Journal of Clinical Pharmacology, EarlyView.
Abstract This review focuses on the development of radiopharmaceutical imaging agents and radioligand therapeutics for paediatric use. Nuclear medicine plays an important role in the diagnosis and treatment of various childhood conditions, including cancers, infections and brain disorders.
Justin L. Hay, Amandine Manon, Hunter Stephens, S. Y. Amy Cheung, Bart de Keizer, Erik T. te Beek   +5 more
wiley   +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

Beyond the label: Rethinking off‐label drug use in paediatrics. Towards a scientifically grounded and safer future for paediatric pharmacotherapy

British Journal of Clinical Pharmacology, EarlyView.
Abstract Despite regulatory progress being made in the past two decades, off‐label drug use in paediatrics remains pervasive, with prevalence estimated between 3% and 97% of prescriptions across different clinical settings. Off‐label use—defined as prescribing outside the conditions described in the Summary of Product Characteristics (SmPC)—is often ...
Tjitske M. van der Zanden, Miriam G. Mooij, Matthijs de Hoog, Saskia N. de Wildt   +3 more
wiley   +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  

Through the lens of marketing authorization holders: experience in use of real‐world data and real‐world evidence in drug development and regulatory submissions in EU

British Journal of Clinical Pharmacology, EarlyView.
Abstract Aim The aim of this study was to assess the general and product‐specific experiences of MAHs use of RWD/RWE in medicines development and in their regulatory submissions, and to explore organizational aspects of MAHs related to RWD/RWE. Methods An electronic survey was conducted, and information collected directly from MAHs.
Sini M. Eskola, Aleksandar Gigov, Hubertus G. M. Leufkens, Andrew Bate, Marie Louise De Bruin, Helga Gardarsdottir   +5 more
wiley   +1 more source

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

Do drugs approved via expedited approval pathways have therapeutic advantages? A systematic review and meta‐analysis

British Journal of Clinical Pharmacology, EarlyView.
Regulators use expedited approval pathways to speed market approval and patient access to promising new drugs. However, there is uncertainty about whether these pathways are successful in approving drugs with significant therapeutic advantages. This systematic review aims to examine the safety, effectiveness and cost‐effectiveness of drugs approved via
Ashleigh Hooimeyer, Eliza J. McEwin, Zhicheng Wang, Joel Lexchin, Barbara Mintzes   +4 more
wiley   +1 more source

Advance price or purchase commitments to create markets for treatments for diseases of poverty: lessons from three policies

Bulletin of the World Health Organization, 2005
New drugs and vaccines are needed for tackling diseases of poverty in low- and middle-income countries. The lack of effective demand or market for these products translates into insufficient investment being made in research and development to meet the ...
Towse Adrian, Kettler Hannah
doaj  

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