Results 21 to 30 of about 1,727 (105)

Surrogate Endpoints in Regulatory Decision‐Making

Clinical and Translational Science, Volume 18, Issue 12, December 2025.
ABSTRACT To support approval, FDA requires substantial evidence of effectiveness that demonstrates a drug improves meaningful clinical outcomes as measured by how a patient feels, functions, or survives. Effectiveness is measured directly (e.g., by patient‐reported outcome or other clinical outcome assessment) or indirectly (i.e., by use of a surrogate
Linda J. B. Jeng, Jeffrey Siegel
wiley   +1 more source

Progressive Soft Tissue Swelling in a Pediatric Patient Leading to the Diagnosis of Fibrodysplasia Ossificans Progressiva: A Case Report

Pediatric Dermatology, Volume 42, Issue 6, Page 1252-1254, November/December 2025.
ABSTRACT Fibrodysplasia ossificans progressiva (FOP) is a rare genetic disorder characterized by progressive heterotopic ossification (HO) and congenital malformation of the great toes. This case describes a 5‐year‐old Caucasian girl who initially presented with painless neck and back swelling as well as severe limitation of movement in the neck and ...
Orhan Yilmaz, Loretta Fiorillo
wiley   +1 more source

A Retinoid Antagonist Attenuates Bone Growth Inhibition Caused by Growth Plate Injury in Mice

Journal of Orthopaedic Research, Volume 43, Issue 10, Page 1724-1735, October 2025.
ABSTRACT Growth plate injury, which may trigger growth plate fusion or arrest, is a common occurrence in pediatric orthopaedics that can lead to angulation of the limb and limb length discrepancy. Growth plate injuries are currently treated palliatively, with surgical intervention when severe limb length discrepancy or angulation is found during follow‐
Masatake Matsuoka, Ningfeng Tang, Tian Hongying, Kenta Uchibe, Satoru Otsuru, Norimasa Iwasaki, Joshua M. Abzug, Motomi Enomoto‐Iwamoto, Masahiro Iwamoto   +8 more
wiley   +1 more source

Diagnosis of Pediatric Myositis Ossificans Based on Cytomorphology and Molecular Analysis From FNAB Sample: A Case Report

Diagnostic Cytopathology, Volume 53, Issue 7, Page E138-E143, July 2025.
ABSTRACT Myositis ossificans (MO) is a benign soft tissue lesion, characterized by ectopic ossification due to inappropriate fibroblast differentiation, most commonly affecting skeletal muscles. It often occurs in young adults after muscle trauma, predominantly in male patients and very rarely in children. We describe the case of a previously healthy 3‐
Živa Ledinek, Milica Stefanović, Blaž Mavčič, Maja Česen Mazić, Ana Gazikalović, Daja Šekoranja, Simona Miceska, Veronika Kloboves Prevodnik   +7 more
wiley   +1 more source

Post‐COVID‐19 Exacerbation of a Stable Fibrous Dysplasia: A Case Report

Clinical Case Reports, Volume 13, Issue 4, April 2025.
ABSTRACT Fibrous dysplasia (FD) is a rare, benign fibro‐osseous lesion characterized by replacement of normal bone with extensive fibrous stroma due to abnormalities in osteoblast differentiation. After puberty and during adulthood, FD lesions usually become quiescent. However, some cases show signs of regrowth and reactivation.
Mohammed Taib Fatih, Mohammed Abdalla Mahmood, Mohammed Khalid Mahmood, Amanj Ismael Tahir, Handren Ameer Kurda, Mohammed Aso Abdulghafor, Balen Hamid Qadir, Zana Fuad Noori   +7 more
wiley   +1 more source

Genetic Testing Utilization in the U.S. Registry for Childhood Interstitial and Diffuse Lung Diseases

Pediatric Pulmonology, Volume 60, Issue 4, April 2025.
ABSTRACT Introduction Childhood interstitial and diffuse lung diseases (chILD) comprise a diverse group of rare disorders. Identifying the underlying cause is crucial for treatment, prognosis, and estimating recurrence risk. The objective of this study was to assess the utilization of genetic testing for subjects enrolled in the United States National ...
Laura A. Voss, Rebekah J. Nevel, Jennifer A. Wambach, Lawrence M. Nogee, Robin R. Deterding, Alicia M. Casey, Michael G. O'Connor, Daniel I. Craven, Jane B. Taylor, Gail H. Deutsch, Jade B. Tam‐Williams, Lea C. Steffes, Steven K. Brennan, Maria T. Santiago, Sara C. Sadreameli, Andrea F. Heras, Michael R. Powers, Antonia P. Popova, Manvi Bansal, Aaron Hamvas, William A. Gower, Fernando Urrego, Lisa R. Young, for The ChILD Registry Collaborative   +23 more
wiley   +1 more source

Future of fibrodysplasia ossificans progressiva management: A systematic review of investigational therapies

Rheumatology &Autoimmunity, Volume 5, Issue 1, Page 28-36, March 2025.
Fibrodysplasia ossificans progressiva, a rare genetic disorder caused by ACVR1 mutations, leads to soft tissue ossification. Recent advancements, including food and drug administration‐approved palovarotene, show promise despite safety concerns. Therapies like tofacitinib and imatinib demonstrate potential, but larger, controlled trials are essential ...
Muhammad Ikrama, Muhammad Usama, Shifa Israr, Maryam Humayon, Muhammad Umair Javaid, Shafqat Abbas, Muhammad Aliyan Haider   +6 more
wiley   +1 more source

ECSIT‐X4 is Required for Preventing Pressure Overload‐Induced Cardiac Hypertrophy via Regulating Mitochondrial STAT3

Advanced Science, Volume 12, Issue 8, February 24, 2025.
ECSIT‐X4 is the predominant cardiac isoform of ECSIT, encoded by the Ecsit‐X4 transcript variant in adult cardiomyocytes. The splicing junction of exon 9 generates the mouse Ecsit‐X4 isoform, resulting in a recognizable C‐terminal sequence. It is shown that ECSIT‐X4 interacts with STAT3, enhancing the activity of complex I, thereby promoting ...
Xia Lu, Tingting Tong, Haoliang Sun, Yi Chen, Yongfeng Shao, Pengxi Shi, Linli Que, Li Liu, Guoqing Zhu, Qi Chen, Chuanfu Li, Jiantao Li, Shuo Yang, Yuehua Li   +13 more
wiley   +1 more source

Fibrodysplasia Ossificans Progressiva

JCR: Journal of Clinical Rheumatology, 2015
Aniket N, Tavare, Jane, Young
openaire   +3 more sources

Issue Information

Rheumatology &Autoimmunity, Volume 5, Issue 1, Page 1-4, March 2025.
wiley   +1 more source