Results 181 to 190 of about 32,298 (248)

Exploring the Burden on Patients Living With and Receiving Treatment for Immune Thrombocytopenia (ITP): Patient and Physician Perceptions From the ITP World Impact Survey (I‐WISh) 2.0

American Journal of Hematology, Volume 101, Issue 8, Page 1954-1968, August 2026.
ABSTRACT Limited data exist on how patients and physicians perceive immune thrombocytopenia (ITP) symptoms and treatment‐related burden. I‐WISh (ITP World Impact Survey) 2.0 surveyed 1018 patients and 431 physicians in 15 countries to characterize the impact of ITP and its treatments on patients.
Nichola Cooper, James Bussel, Waleed Ghanima, Drew Provan, Yoshiaki Tomiyama, Ming Hou, Donald M. Arnold, Cristina Santoro, Francesco Zaja, Barbara Lovrencic, Mervyn Morgan, Michal Winograd, Jennifer DiRaimo, Danielle Boyle, Olivera Rajkovic‐Hooley, Meritxell Vendranas, Susan Frade, Caroline Kruse   +17 more
wiley   +1 more source

Availability and Access to Orphan Drugs for Rare Cancers in Bulgaria: Analysis of Delays and Public Expenditures. [PDF]

Cancers (Basel)
Kostadinov K, Popova-Sotirova I, Marinova Y, Musurlieva N, Iskrov G, Stefanov R.   +5 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

Therapeutic Value of Orphan Drugs Approved by Health Canada: A Cross-Sectional Study. [PDF]

J Gen Intern Med
Lexchin J.
europepmc   +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

A Diagnostic Dilemma: Hypophosphatemic Rickets Unmasking Tyrosinemia Type 1: A Case Report

Clinical Case Reports, Volume 14, Issue 8, August 2026.
ABSTRACT A 7.5‐year‐old Pakistani girl was misdiagnosed with hypophosphatemic rickets. Progressive skeletal deformities, hepatomegaly, and renal tubular dysfunction were detected despite standard treatment. Due to the atypical findings, genetic testing was performed and confirmed the diagnosis of Hereditary tyrosinemia Type 1.
Muhammad Wajid Siddique, Muhammad Hanzla Shahzad, Mifrah Rahat Khan, FNU Mahparah, Kanchan kumari, Muhammad Husnain Ahmad   +5 more
wiley   +1 more source

Sustained Effectiveness of Lanadelumab in Preventing Hereditary Angioedema Attacks: The ENABLE Study

Clinical and Translational Allergy, Volume 16, Issue 8, August 2026.
ABSTRACT Background Lanadelumab has been approved for hereditary angioedema (HAE) long‐term prophylaxis since 2018. The Phase 4, prospective ENABLE Study (NCT04130191) evaluated the long‐term effectiveness and safety of lanadelumab in clinical practice across Europe and the Middle East.
Andrea Zanichelli, Walter A. Wuillemin, Markus Magerl, Andreas Recke, Mauro Cancian, Emel Aygören‐Pürsün, Ramón Lleonart Bellfill, Aharon Kessel, Tamar Kinaciyan, Robin Lochbaum, Mona Al‐Ahmad, Irmgard Andresen, Maureen Watt, Natalie Khutoryansky, Daniel N. Castaner, Inmaculada Martinez‐Saguer   +15 more
wiley   +1 more source

The impact of whole lung irradiation in lung metastatic rhabdomyosarcoma: A pooled analysis of two European trials and one European registry

Cancer, Volume 132, Issue 15, 1 August 2026.
ABSTRACT Background Lung metastases in patients with metastatic rhabdomyosarcoma (RMS) have not been treated uniformly across Europe. This provides comparison of the impact of whole lung irradiation (WLI). Methods Lung‐metastatic patients included in the Cooperative Weichteilsarkom Studiengruppe‐IV 2002, European Pediatric Soft Tissue Sarcoma Study ...
Amadeus T. Heinz, Philip Heesen, Johannes H. M. Merks, Véronique Minard‐Colin, Martin Ebinger, Alison L. Cameron, Semi Harrabi, Henry Mandeville, Reineke A. Schoot, Jörg Fuchs, Gabriela Guillen, Ewa Koscielniak, Julia C. Chisholm, Monika Sparber‐Sauer, Gianni Bisogno   +14 more
wiley   +1 more source

A Systematic Review on Disease‐Modifying Therapies in Parkinsonian Disorders

Clinical Pharmacology &Therapeutics, Volume 120, Issue 2, Page 357-374, August 2026.
Parkinsonian disorders, including Parkinson's disease, Lewy body dementia, multiple system atrophy, and progressive supranuclear palsy, are progressive neurodegenerative conditions with no treatment options to slow disease progression. This systematic review provides an overview of evidence of disease‐modifying therapies that have been evaluated in ...
Pepijn P.N.M. Eijsvogel, Lars M.G. Smits, Philip H.C. Kremer, Geert Jan Groeneveld   +3 more
wiley   +1 more source

A Tale of Two Mechanisms: The p53 Modulator COTI‐2 Is a Zn Metallochaperone

Drug Development Research, Volume 87, Issue 5, August 2026.
ABSTRACT Mutations in, or misregulation of, Tp53 are found in approximately 50% of all cancers. p53 functions by ensuring that cells with irretrievably damaged DNA undergo apoptosis. Tp53 mutations often induce conformational changes that inhibit activity; however, small‐molecule chaperones could theoretically restore conformation and activity. COTI‐2,
İrem Şimşek, Farsheed Shahbazi‐Raz, Michael J. Krause, Azam Mohammadzadeh, Maryam Kosar, Peihan Xu, Samra Khan, Olena Tykhoniuk, Ashley DaDalt, Deya'a Almasri, Lara K. Watanabe, Kaitlyn Breault, John J. Hayward, Fraser S. Pick, Richard L. Ho, Jeremy M. Rawson, John F. Trant   +16 more
wiley   +1 more source