Results 101 to 110 of about 13,796 (248)

Unveiling the Gray: A Rare Case of Gray Platelet Syndrome With Hepatomegaly and Immune Dysregulation in a 14-Year-Old

Case Reports in Hematology
Gray platelet syndrome (GPS) is a rare inherited platelet disorder characterized by the presence of gray platelets on blood smears, resulting from a deficiency of α-granules.
Muhammad Takhman, Moath Hattab, Reem Shihab, Asmaa Sarama, Sultan Mosleh   +4 more
doaj   +1 more source

Clinical Experience With Eliglustat in Children With Gaucher Disease Type 1

American Journal of Medical Genetics Part A, EarlyView.
Erika R. Vucko, Allegra Quadri, Rachel E. Hickey, Carly A. Rasmussen, Monika Izdebski, Joel Charrow, Carlos E. Prada   +6 more
wiley   +1 more source

Cost‐effectiveness of DPYD genotyping prior to fluoropyrimidine‐based treatment for colorectal cancer in Wales

British Journal of Clinical Pharmacology, EarlyView.
Abstract Background Regulatory guidance in the United Kingdom advises DPYD genotyping prior to fluoropyrimidine‐based treatment. This economic evaluation estimated the costs and outcomes associated with DPYD screening prior to prescribing fluoropyrimidines for colorectal cancer in Wales and also considers additional variants to those included in ...
Catrin O. Plumpton, Sian M. Morgan, Richard A. Adams, Samantha Cox, Angharad A. Rudkin, Megan Fealey, Craig Barrington, Michelle D. Wood, Dyfrig A. Hughes   +8 more
wiley   +1 more source

Model‐informed optimal dose selection of golcadomide in combination with R‐CHOP in patients with previously untreated aggressive B‐cell lymphoma

British Journal of Clinical Pharmacology, EarlyView.
Aims Golcadomide is a potential first‐in‐class oral CELMoD™ agent for the treatment of lymphoma. This study aims to develop model‐informed drug development (MIDD) approaches to support optimal dose selection of golcadomide in combination with immunotherapy rituximab, cyclophosphamide, doxorubicin, vincristine and prednisone (R‐CHOP) in alignment with ...
Xirong Zheng, Zhiling Yu, Mark Kaplan, Argyrios Gkasiamis, Akshay Sudhindra, Li Zhu, Manisha Lamba, Fan Wu   +7 more
wiley   +1 more source

Spontaneous splenic rupture due to extramedullary haematopoiesis in a patient with inherited thrombocytopenia. [PDF]

Blood Transfus, 2021
Zaninetti C, Sacco C, Rahal D, Barozzi S, Balduini CL, Lodigiani C, Pecci A.   +6 more
europepmc   +1 more source

Isoform Specificity of a Compound Targeting Actin Filaments Containing Tropomyosin Tpm1.8/1.9

Cytoskeleton, EarlyView.
ABSTRACT The unbranched actin filaments in mammalian cells are usually composed of co‐polymers of a specific tropomyosin isoform with actin. Genetic manipulation has revealed that the tropomyosins largely define the functional properties of actin filaments in an isoform‐specific, non‐redundant manner.
Jeff Hook, Edna C. Hardeman, Peter W. Gunning   +2 more
wiley   +1 more source

Thrombocytopenia in patients with inborn errors of immunity

BMC Immunology
Background Inborn errors of immunity (IEI) are inherited defects of innate or adaptive immune system. Thrombocytopenia is a significant multifactorial complication in IEI patients leading to severe clinical consequences including coagulative disorders ...
Saba Fekrvand, Maryam Mohtashami, Negin Sanadgol, Helia Salehi, Najmeh Nameh Goshay Fard, Ehsan Khoshnezhad Afkham, Zahra Chavoshzadeh, Nima Parvaneh, Seyed Alireza Mahdaviani, Samin Sharafian, Sahar Barzamini, Hamid Ahanchian, Arash Kalantari, Alireza Shafiei, Marzieh Tavakol, Farhad Abolnezhadian, Mina Kianmanesh Rad, Gholamreza Hassanpour, Taher Cheraghi, Amir Salehi Farid, Samaneh Delavari, Hassan Abolhassani, Nima Rezaei, Reza Yazdani   +23 more
doaj   +1 more source

The Copenhagen founder variant GP1BA c.58T>G is the most frequent cause of inherited thrombocytopenia in Denmark. [PDF]

J Thromb Haemost, 2021
Leinøe E, Brøns N, Rasmussen AØ, Gabrielaite M, Zaninetti C, Palankar R, Zetterberg E, Rosthøj S, Ostrowski SR, Rossing M.   +9 more
europepmc   +1 more source

Virtual Twin Approach Using Physiologically Based Pharmacokinetic Modeling to Support Precision Dosing of Valproic Acid in Geriatric Patients

Clinical Pharmacology &Therapeutics, EarlyView.
Personalized dosing is particularly important for drugs with narrow therapeutic indices in geriatric patients, who exhibit substantial physiological variability and limited pharmacokinetic (PK) evidence to guide individualized dose selection. Valproic acid (VPA) is an effective treatment option for bipolar disorder in older adults, yet dosing largely ...
Yoo Jin Jang, Dong‐Gyu Heo, Eunjin Hong   +2 more
wiley   +1 more source

A Practical Guide to Implementing Real‐World Evidence as Primary Basis for Approval and Label Expansion

Clinical Pharmacology &Therapeutics, EarlyView.
In June 2026, the FDA issued a revised draft guidance describing the circumstances in which one adequate and well‐controlled clinical investigation plus confirmatory evidence may satisfy the statutory substantial evidence standard for establishing efficacy and safety. Confirmatory evidence includes real‐world evidence (RWE).
Tracy J. Mayne, Maurice A. Brookhart, Nancy A. Dreyer   +2 more
wiley   +1 more source