Results 261 to 270 of about 3,868,024 (353)

Excluding Orphan Drugs from the 340B Drug Discount Program: the Impact on 18 Critical Access Hospitals

, 2012
Madeline Carpinelli Wallack, Todd D. Sorensen   +1 more
openalex   +2 more sources

Sources of Information and Pharmacists' Knowledge Regarding Rare Diseases and Orphan Drugs: Cross-Sectional Study In Serbia [PDF]

, 2014
Jasmina Arsić, Dušanka Krajnović, Ljiljana Tasić, Valentina Marinković, Jelena Djordjevic   +4 more
openalex   +1 more source

Orphan drugs: Indian perspective

Indian Journal of Pharmacology, 2017
Harish Kumar, Bikash Medhi, Phulen Sarma
openaire   +3 more sources

Mesenchymal Stromal Cell Secretome and Its Key Bioactive Metabolites Induce Long‐Term Neuroprotection After Traumatic Brain Injury in Mice

Advanced Science, Volume 12, Issue 29, August 7, 2025.
Pischiutta F and coworkers identify prostaglandins and kynurenine as key mediators of the mesenchymal stromal cells‐derived secretome's neuroprotective effects. A synthetic cocktail composed of these factors promotes long‐term functional recovery, reduces brain structural damage, and modulates neuroinflammation after traumatic brain injury, supporting ...
Francesca Pischiutta, Francesca Tribuzio, Marta Magatti, Giulia De Simone, Federico Moro, Giovanni Nattino, Fabiola Signorini, Luther Loose, Enrico Caruso, Costanza Bertani, Edoardo Mazzone, Rosaria Pascente, Edoardo Micotti, Antonietta Rosa Silini, Fabrizio Ortolano, Maria Chiara Trolese, Marco Bolis, Luca Guarrera, Martina Bruna Violatto, Paolo Bigini, Cristina Banfi, Roberta Pastorelli, Ornella Parolini, Laura Brunelli, Elisa R Zanier   +24 more
wiley   +1 more source

The Orphan Drug Act at 40: Legislative Triumph and the Challenges of Success. [PDF]

Milbank Q
Saltonstall P, Ross H, Kim PT.
europepmc   +1 more source

Effects of Secukinumab on Enthesiophyte and Erosion Progression in Psoriatic Arthritis: A One‐Year Double‐Blind, Randomized, Placebo‐Controlled Trial Using High‐Resolution Peripheral Quantitative Computed Tomography

Arthritis &Rheumatology, Volume 77, Issue 8, Page 1015-1025, August 2025.
Objective This study aimed to ascertain the effect of secukinumab on erosion and enthesiophyte progression in psoriatic arthritis (PsA) by using high‐resolution peripheral quantitative computed tomography (HR‐pQCT). Methods This was a phase 4, double‐blind, randomized, placebo‐controlled trial.
Yingzhao Jin, Isaac T. Cheng, Ho So, Billy T. Lai, Shirley K. Ying, Kitty Y. Kwok, James Griffith, Vivian Hung, Cheuk‐Chun Szeto, Jack J. Lee, Ling Qin, Lai‐Shan Tam   +11 more
wiley   +1 more source

Real‐World Data From a Molecular Tumor Board‐Assisted Cancer Care From a Single Center in The Czech Republic: Is Precision Oncology an Accessible Option, or a Privilege for a Minority of Patients?

Cancer Medicine, Volume 14, Issue 15, August 2025.
This study presents a real‐world analysis of patients with advanced solid tumors discussed at the Molecular Tumor Board (MTB) of University Hospital Brno, Czech Republic, between 2021 and 2025. Comprehensive genomic profiling guided matched therapies in 17.4% of cases, with a clinical benefit (PFS2/PFS1 ≥ 1.3) observed in 41.4% of evaluable patients ...
Michal Eid, Markéta Bednaříková, Jakub Vlažný, Jitka Hausnerová, Renata Taslerová, Sára Vilmanová, Martina Jelínková, Alena Homolová, Martin Gryc, Jakub Trizuljak, Zdeněk Pavlovský, Štěpán Tuček, Dagmar Brančíková, Monika Bratová, Tomáš Rohan, Zdeněk Kala, Zdeněk Král, Jiří Mayer, Adam Svobodník, Ondřej Slabý   +19 more
wiley   +1 more source

Access to orphan drugs in the Middle East: Challenge and perspective

, 2012
Ziyad S. Almalki
openalex   +2 more sources

Challenges in Measuring the Societal Value of Orphan Drugs: Insights from a Canadian Stated Preference Survey [PDF]

, 2015
Nick Dragojlovic, Shirin Rizzardo, Nick Bansback, Craig Mitton, Carlo A. Marra, Larry D. Lynd   +5 more
openalex   +1 more source

FDA Approval of Orphan Drug Indications for Pediatric Patients, 2011-2023.

JAMA Pediatr
Kakkilaya A, Shahzad M, Bourgeois FT.
europepmc   +1 more source