Results 61 to 70 of about 1,419,402 (298)

Disease awareness or subtle product placement? Orphan diseases featured in the television series “House, M.D.” - a cross-sectional analysis

BMC Medical Ethics, 2020
Background Approximately 7% of the general population is affected by an orphan disease, which, in the United States, is defined as affecting fewer than 1 in 1500 people. Disease awareness is often low and time-to-diagnosis delayed. Different legislations
Konstantin Mechler, Juliane Rausch, William K. Mountford, Markus Ries   +3 more
doaj   +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

Efficacy and Safety of Nanoencapsulated Sirolimus Plus Pegadricase: Results From the Randomized, Placebo‐Controlled Phase 3 Trials

Arthritis &Rheumatology, EarlyView.
Objective DISSOLVE I and II examined the efficacy and safety of nanoencapsulated sirolimus (NAS) plus pegadricase (NASP) in patients with uncontrolled gout (UG). Methods In these double‐blind, placebo‐controlled Phase 3 trials of NASP, patients were randomized 1:1:1 to infusions of high‐dose (HD) or low‐dose (LD) NAS plus pegadricase (HD NASP, LD NASP,
Herbert S. B. Baraf, Puja P. Khanna, Milan Petronijevic, Mamuka Lortkipanidze, Anand Patel, Kwabena Ayesu, Michael H. Pillinger, Atul Singhal, Joanna Sobierska, Jacquie Christie, Peter Traber, Rehan Azeem, Wesley DeHaan, Hugues Santin‐Janin, Bhavisha Desai, Alan Kivitz   +15 more
wiley   +1 more source

Raskin, Hy (Box 91, Folder 8) [PDF]

, 1976
Series VIII: Audiocassettes features informal oral histories about the Hebrew Orphan Asylum and its alumni.Digital recordingDigital finding ...
Hebrew Orphan Asylum of New York
core  

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

Siegel, Harry and Sally (Box 91, Folder 16) [PDF]

, 1987
Series VIII: Audiocassettes features informal oral histories about the Hebrew Orphan Asylum and its alumni.Digital recordingDigital finding ...
Hebrew Orphan Asylum of New York
core  

Thirty Years of Orphan Drug Legislation and the Development of Drugs to Treat Rare Seizure Conditions: A Cross Sectional Analysis.

PLoS ONE, 2016
BackgroundEpilepsy is a serious chronic health condition with a high morbidity impairing the life of patients and afflicted families. Many epileptic conditions, especially those affecting children, are rare disorders generating an urgent medical need for
Jan Henje Döring, Anette Lampert, Georg F Hoffmann, Markus Ries   +3 more
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

White, Ed (Box 91, Folder 23) [PDF]

, 1977
Series VIII: Audiocassettes features informal oral histories about the Hebrew Orphan Asylum and its alumni.Digital recordingDigital finding ...
Hebrew Orphan Asylum of New York
core  

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