Results 41 to 50 of about 22,997 (249)

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 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

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

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

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

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

Facilitating Expedited Drug Approval—the Past, Present, and Future of Clinical Trials for Pediatric Rheumatic Disease

Arthritis Care &Research, EarlyView.
Pamela F. Weiss, Hermine I. Brunner
wiley   +1 more source

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

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

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
Objectives The 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.
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