Results 121 to 130 of about 32,298 (248)

Negotiating Medical Insurance Drug Prices: The Role in Reducing Costs of Orphan Drugs for Rare Diseases. [PDF]

Int J Health Policy Manag, 2023
Lu J, Ying X, Li Z.
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

Informing Dose for Pediatric Rare Diseases-A Survey of Recent Orphan Drugs Approvals. [PDF]

Clin Transl Sci
Fletcher EP, Kim C, Mei H, AbuAsal B, Samuels S, Madabushi R, Ramamoorthy A.   +6 more
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

The impact of China's drug regulatory reform on access to orphan drugs: a cross-sectional study of diseases listed in the rare disease catalog. [PDF]

Front Pharmacol
Lan Y, Lin X, Huang Z, Zan W.
europepmc   +1 more source

Evidence That the Protein Phosphatase Activity of PTEN Contributes to Embryonic Development and Tumor Suppression

Cancer Science, EarlyView.
Mice expressing only mutant PTEN Y138L, a protein which shows normal suppression of cellular AKT yet lacks protein phosphatase activity, die in utero, and heterozygous mice display a range of tumors. This indicates both the lipid and protein phosphatase activities of PTEN work together for normal tumor suppression and embryonic development.
Priyanka Tibarewal, Laura Spinelli, Nisha Kriplani, Helen Wise, Nadege Poncet, Giulia Marzano, Karen E. Anderson, Katarzyna M. Grzes, Zofia Varyova, Mahreen Adil, C. Peter Downes, Phillip T. Hawkins, Len R. Stephens, Kate G. Storey, Doreen A. Cantrell, Bart Vanhaesebroeck, Nicholas R. Leslie   +16 more
wiley   +1 more source

Access to orphan drugs in adults with inherited metabolic diseases in Switzerland: a single-center retrospective cohort study. [PDF]

Orphanet J Rare Dis
Antoni G, Kumps C, Berger J, Barman-Aksözen J, Tran C.   +4 more
europepmc   +1 more source

Lifecycle management of orphan drugs approved in Japan. [PDF]

Orphanet J Rare Dis, 2022
Seki K, Suzuki H, Abe S, Saotome C.
europepmc   +1 more source

Clinical and genetic characterization of intellectual disability

Developmental Medicine &Child Neurology, EarlyView.
This study examines the etiological factors and comorbidities in a large cohort of Finnish patients with intellectual disability. Genetic causes—including chromosomal abnormalities and pathogenic gene variants—were more frequently identified in individuals with moderate to profound intellectual disability.
Aarni Venetvaara, Minna Kraatari‐Tiri, Jussi‐Pekka Tolonen, Marko Merikukka, IDGEN Study Group, Hannaleena Kokkonen, Teija Paakkola, Susanna Varis, Antti Dahl, Jarmo Körkkö, Jonna Komulainen‐Ebrahim, Jukka Moilanen, Johanna Uusimaa, Outi Kuismin, Elisa Rahikkala   +14 more
wiley   +1 more source

Orphan Drugs and Orphan Tests in the USA

Public Health Genomics, 2004
openaire   +2 more sources