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Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma^®) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System. [PDF]

Pharmaceuticals (Basel)
Ruggiero R, Balzano N, Nicoletti MM, di Mauro G, Fraenza F, Campitiello MR, Rossi F, Capuano A.   +7 more
europepmc   +1 more source

From cramming to binge‐watching: Integrating documentary‐based assessment into a pharmacology and toxicology curriculum—a qualitative study

British Journal of Clinical Pharmacology, Volume 92, Issue 8, Page 2474-2486, August 2026.
Abstract Aims In the context of pharmacology and toxicology education, there is a growing shift toward programmatic assessment models that prioritize longitudinal learning, reflection and development of higher‐order cognitive skills. As part of this transition, we are exploring alternative and more meaningful forms of assessment. This qualitative study
Narin Akrawi, Heleen van der Sijs, Floor van Rosse   +2 more
wiley   +1 more source

Advanced therapy medicinal products (ATMPs) – An analysis of the global regulatory status

British Journal of Clinical Pharmacology, Volume 92, Issue 8, Page 2909-2916, August 2026.
Abstract Background A rapid progress in the development of advanced therapy medicinal products (ATMPs) has taken place over the past decades. The ATMPs hold great potential but face challenges in the clinical development and the regulatory process.
Allan Cramer, Freja Karuna Hemmingsen Sørup, Marie Lund, Tonny Studsgaard Petersen, Kristian Karstoft   +4 more
wiley   +1 more source

Intravital microscopy: An innovative approach to track real‐time muscle integrity and pathology in Pompe disease

Clinical and Translational Discovery
Nishitha R. Pillai, Reena V. Kartha
doaj   +1 more source

Abstracts

Molecular Oncology, Volume 20, Issue S1, Page 1-692, August 2026.
Abstracts submitted to the ‘EACR 2026 Congress: Innovative Cancer Science’, from 08–11 June 2026 and accepted by the Congress Organising Committee are published in this Supplement of Molecular Oncology, an affiliated journal of the European Association for Cancer Research (EACR).
wiley   +1 more source

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Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU

European Journal of Clinical Pharmacology, 2012
To assess the methodological quality of Orphan Medicinal Product (OMP) dossiers and discuss possible reasons for the small number of products licensed.Information about orphan drug designation, approval, refusal or withdrawal was obtained from the website of the European Medicines Agency and from the European Public Assessment Reports.From 2000 up to ...
Vittorio Bertele', Roberta Joppi, Bertele' Vittorio   +2 more
exaly   +3 more sources

Orphan Drugs in Oncology

2018
Rare diseases represent a group of conditions affecting a very limited number of patients. Low profitability resulting from the small size of target population coupled with difficulties in conducting the research causes the lack of interest from the pharmaceutical industry.
Daria, Korchagina, Szymon, Jaroslawski, Guy, Jadot, Mondher, Toumi   +3 more
openaire   +2 more sources