Results 181 to 190 of about 3,868,024 (353)

Does Misery Love Company? Evidence from pharmaceutical markets before and after the Orphan Drug Act

, 2003
Frank R. Lichtenberg, Joel Waldfogel
openalex   +1 more source

Role of Histone Deacetylase and Inhibitors in Cardiovascular Diseases

Cell Proliferation, EarlyView.
HDACs play an important role in the occurrence and development of cardiovascular diseases, such as myocardial hypertrophy, hypertension, and atherosclerosis. HDAC inhibitors have broad prospects for the treatment of CVD, and different HDAC subtypes can act through different mechanisms.
Li‐Ying Zhang, Yue‐Yue Wang, Ri Wen, Tie‐Ning Zhang, Ni Yang   +4 more
wiley   +1 more source

Age at onset and gene variants predict lifespan and disease duration in childhood neuronal ceroid lipofuscinoses

Developmental Medicine &Child Neurology, EarlyView.
Abstract Aim To address disease progression in a cohort of patients with childhood‐onset neuronal ceroid lipofuscinosis (NCL), a group of genetic disorders leading to progressive dementia. Method In this retrospective study, selected clinical features (age at onset, at death, and disease duration) and pathogenicity of individual genotypes were selected
Alessandro Simonati, Francesco Pezzini, Nardo Nardocci, CLNet Consortium, C Aiello, R Battini, R Biancheri, M Bonsignore, R Carrozzo, B Dalla Bernardina, S Della Vecchia, S Doccini, A Donati, M Filocamo, B Garavaglia, T Granata, S Grosso, AM Laverda, V Leuzzi, C Marini, M Mastrangelo, T Messena, M Salandin, S Sartori, N Specchio, P Striano, F Taioli, A Tessa, E Veneselli, Filippo M. Santorelli   +29 more
wiley   +1 more source

Alert Card for Patients on Biologicals and Immunosuppressives: An EAACI Position Paper

Allergy, EarlyView.
Adam Chaker, Paula Kauppi, Sevim Bavbek, Apostolos Bossios, Alexia Chatzipetrou, Davide Firinu, Barbara Rogala, Andrea Matucci, Alessandra Vultaggio, Petr Panzner, Wojciech Feleszko, Ron Eliashar, Ioanna Agache, Ludger Klimek, Maria Torres, Oliver Pfaar, Oscar Palomares, Mohamed Shamji, Thomas Eiwegger, Antonios G. A. Kolios   +19 more
wiley   +1 more source

Recent advances in multimodality imaging‐guided therapy in pericarditis

European Journal of Clinical Investigation, EarlyView.
Multiple imaging targets have been identified as essential biomarkers to confirm pericardial thickening, edema, neovascularization, inflammation, fibrosis/calcification, cardiac tamponade physiology, and constriction. This novel approach is called imaging‐guided therapy, which guides anti‐inflammatory therapy, assesses treatment response, assists in ...
Joseph El Roumi, Aldo L. Schenone, Paul Cremer, Tom Kai Ming Wang, Allan Klein   +4 more
wiley   +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

Autoimmunity in MASLD: Focus on autoantibodies, anti‐apolipoprotein A1 IgG and G protein‐coupled receptors

European Journal of Clinical Investigation, EarlyView.
This review outlines the potential role of anti‐GPCR autoantibodies in MASLD/MASH, given their involvement in a wide range of diseases and their possible contribution to MASLD/MASH pathophysiology. We also focused on anti‐ApoA1 autoantibodies, sharing functional features with anti‐GPCR autoantibodies and have been shown to promote liver steatosis in ...
Sabrina Pagano, Emmanuel Somm, François R. Jornayvaz, Nicolas Vuilleumier   +3 more
wiley   +1 more source

How do patients and other members of the public engage with the orphan drug development? A narrative qualitative synthesis. [PDF]

Orphanet J Rare Dis, 2023
Frost J, Hall A, Taylor E, Lines S, Mandizha J, Pope C.   +5 more
europepmc   +1 more source

PHP39 PUBLIC FUNDING OF DRUGS FOR ORPHAN DISEASES

, 2005
Dyfrig Hughes
openalex   +1 more source

Final Results From a Large, Non‐Interventional, Phase 4 Study of Ruxolitinib for the Treatment of Myelofibrosis in Clinical Routine

European Journal of Haematology, EarlyView.
ABSTRACT JAKoMo was a long‐term, multicenter, non‐interventional study observing the efficacy, safety, and quality of life (QOL) effects of ruxolitinib (RUX), managed per clinical routine at investigator discretion, for treatment of 943 patients with myelofibrosis (MF) in 122 German centers.
Steffen Koschmieder, Susanne Isfort, Clemens Schulte, Lutz Jacobasch, Thomas Geer, Marcel Reiser, Michael Koenigsmann, Bernhard Heinrich, Jürgen Wehmeyer, Eyck von der Heyde, Hans Tesch, Benedikt Gröschl, Petra Bachhuber, Susanne Großer, Michael Koehler, Heike L. Pahl   +15 more
wiley   +1 more source