Results 161 to 170 of about 1,419,402 (298)

Novel approaches for drug development against chronic primary pain: A systematic review

British Journal of Pharmacology, EarlyView.
Abstract Chronic primary pain (CPP) persisting for more than 3 months, associated with significant emotional distress without any known underlying cause, is an unmet medical need. Traditional or adjuvant analgesics do not provide satisfactory pain relief for a great proportion of these patients.
Valéria Tékus, Éva Borbély, Andreas Goebel, Ralf Baron, Zsófia Hajna, Zsuzsanna Helyes   +5 more
wiley   +1 more source

Correction to: Analysis of the first ten years of FDA’s rare pediatric disease priority review voucher program: designations, diseases, and drug development

Orphanet Journal of Rare Diseases
Catherine Mease, Kathleen L. Miller, Lewis J. Fermaglich, Jeanine Best, Gumei Liu, Erika Torjusen   +5 more
doaj   +1 more source

Orphan drug propranolol for infantile hemangioma: ten-year real-world safety data from the FAERS database. [PDF]

Orphanet J Rare Dis
Li X, Zhao X, Yang C, Chen A.
europepmc   +1 more source

Peripheral targets for neuropathic pain

British Journal of Pharmacology, EarlyView.
Neuropathic pain represents a significant clinical challenge, with still limited pharmacological approaches to symptomatic relief. This review focuses on molecular targets implicated in neuropathic pain, particularly those involved in peripheral mechanisms. Using the IUPHAR/BPS database of biological targets, their occurrence together with ‘neuropathic
Amirhossein Afsharipour, Cosmin I. Ciotu, Felix J. Resch, Michael J. M. Fischer   +3 more
wiley   +1 more source

Orphan Drug Approval in Canada, 1999-2022: A Cross-sectional Study. [PDF]

Int J Health Policy Manag
Lexchin J.
europepmc   +1 more source

Acute anaphylactic and multiorgan inflammatory effects of Comirnaty in pigs: evidence of spike protein mRNA transfection and paralleling inflammatory cytokine upregulation

British Journal of Pharmacology, EarlyView.
Abstract Background and Purpose Rare but severe adverse events (AEs) associated with mRNA‐lipid nanoparticle (LNP) COVID‐19 vaccines, such as Comirnaty, include anaphylaxis and inflammatory organ disorders, but their underlying mechanisms are poorly understood. We aimed to develop an animal model to study these phenomena.
László Dézsi, Gábor Kökény, Gábor Szénási, Csaba Révész, Tamás Mészáros, Bálint A. Barta, Réka Facsko, Anna Szilasi, Tamás Bakos, Gergely T. Kozma, Attila B. Dobos, Béla Merkely, Tamás Radovits, János Szebeni   +13 more
wiley   +1 more source

Advances in orphan drug development for alpha-1 antitrypsin deficiency: a 2025 update from the FDA and EMA. [PDF]

Ther Adv Respir Dis
Höger P, Ries M, Olivares Rivera A, Ersöz H, Buschulte K, Fähndrich S, Kontogianni K, Herth F, Trudzinski FC.   +8 more
europepmc   +1 more source

A qualitative study on the views of experts on the social impact of the high-priced orphan drug nusinersen. [PDF]

Explor Res Clin Soc Pharm, 2023
Rosenberg S, Södergård B, Rosenholm JM, Rauha JP.   +3 more
europepmc   +1 more source

Orphan Drugs and Orphan Tests in the USA

Public Health Genomics, 2004
openaire   +2 more sources

Systematic profiling reveals broad G protein coupling and context‐dependent modulation of cyclic AMP by the orphan receptor GPR139

British Journal of Pharmacology, EarlyView.
Background and Purpose G protein‐coupled receptors (GPCRs) are major drug targets, yet many orphan receptors remain poorly characterized. GPR139 has been implicated in CNS disorders, including schizophrenia and depression, but its signalling mechanisms remain unclear.
Boris Trapkov, Hans Bräuner, Alexander Sebastian Hauser   +2 more
wiley   +1 more source