Results 51 to 60 of about 3,792 (182)

Use of genetically modified muscle and fat grafts to repair defects in bone and cartilage [PDF]

, 2009
We report a novel technology for the rapid healing of large osseous and chondral defects, based upon the genetic modification of autologous skeletal muscle and fat grafts.
A Walsh, C Kirker-Head, CH Evans, F-J Liu, FA Saad, JA Hoyland, JW Wells, LC Gerstenfeld, MB Harris, MS Vrahas, null null, O Betz, RM Porter, TA Einhorn, V Betz, V Glatt   +15 more
core   +4 more sources

Evaluation of clinical cardiac safety of zilurgisertib, an activin receptor‐like kinase‐2 (ALK2) inhibitor, in healthy participants

Clinical Pharmacology in Drug Development, Volume 15, Issue 2, February 2026.
Abstract The oral, small molecule inhibitor of activin receptor‐like kinase‐2, zilurgisertib (INCB000928), is under evaluation in fibrodysplasia ossificans progressiva. Cardiac safety was assessed using electrocardiogram (ECG) parameters and a plasma concentration‐heart rate‐corrected QT (C‐QTc) interval analysis of pooled data from single ascending ...
Yan‐ou Yang, Hong Yang, Xing Liu, Xiaohua Gong, Jay Getsy, Kevin Rockich   +5 more
wiley   +1 more source

Clinical Features, Diagnosis and Management of Fibrodysplasia Ossificans Progressiva

罕见病研究, 2023
Fibrodysplasia ossificans progressiva(FOP) is a rare congenital disease characterized by progressive heterotopic ossification, causing severe immobility with multi-system involvement.
ZHAO Junduo, SHEN Jianxiong
doaj   +1 more source

گزارش 1 مورد بيمار مبتلا به فيبروديسپلازی(ميوزيت) اسيفيکان پيشرونده [PDF]

, 2003
فيبروپلازی(ميوزيت) اسيفيکان پيشرونده بيماری نادر بافت همبند می‌باشد که مشخصه آن استخوان‌سازی نابجا در نسج نرم و بطور منتشر در تمام بدن و هالوکس والگوس دوطرفه هيپوپلاستيک(انگشت شست کوچک در پا) می‌باشد.
شيرانی, فاطمه
core  

Patient Retreat in Dose Escalation for Phase I Clinical Trials With Rare Diseases

Statistics in Medicine, Volume 45, Issue 3-5, February 2026.
ABSTRACT Phase I clinical trials aim to identify the maximum tolerated dose (MTD), a task that becomes challenging in rare disease due to limited patient recruitment. Traditional dose‐finding designs, which assign one dose per patient, require a sufficient sample size that may be infeasible for rare disease trials.
Jialu Fang, Guosheng Yin
wiley   +1 more source

An mTOR Signaling Modulator Suppressed Heterotopic Ossification of Fibrodysplasia Ossificans Progressiva

Stem Cell Reports, 2018
Summary: Fibrodysplasia ossificans progressiva (FOP) is a rare and intractable disorder characterized by extraskeletal bone formation through endochondral ossification.
Kyosuke Hino, Chengzhu Zhao, Kazuhiko Horigome, Megumi Nishio, Yasue Okanishi, Sanae Nagata, Shingo Komura, Yasuhiro Yamada, Junya Toguchida, Akira Ohta, Makoto Ikeya   +10 more
doaj   +1 more source

Superior mesenteric artery syndrome in a patient with fibrodysplasia ossificans progressiva

Bone Reports, 2023
An 18-year-old boy with fibrodysplasia ossificans progressiva lost weight at an accelerated rate due to gastrointestinal symptoms, resulting in a weight loss of 36 kg in 1 year.
Tae Young Ahn, Jung Bum Han, Jung Yun Bae, Seung Hun Woo   +3 more
doaj   +1 more source

The Scientific Case for Animal Models: A Perspective From Musculoskeletal Researchers

FASEB BioAdvances, Volume 8, Issue 2, February 2026.
ABSTRACT The National Institutes of Health (NIH) has launched a major initiative to expand human‐based New Approach Methodologies (NAMs) in biomedical research and reduce reliance on animal models. While NAMs offer powerful complementary tools, animal‐based research remains indispensable in musculoskeletal science for understanding complex cellular and
Michael Hadjiargyrou, Blake E. Hildreth III, Frank Ko, Uma Sankar, Tao Yang   +4 more
wiley   +1 more source

Fibrodysplasia Ossificans Progressiva: Literature Review and Case Report

Вопросы современной педиатрии, 2023
Background. Fibrodysplasia ossificans progressiva (FOP) is a genetic disease of the heterotopic ossification group associated with the mutation in ACVR1/ALK2 gene.
Natalya N. Korableva, Evgeniy V. Berestnev, Sergey M. Kiselyov, Natalya F. Chipsanova   +3 more
doaj   +1 more source

Fibrodysplasia Ossificans Progressiva: Case Report. [PDF]

, 2015
Fibrodysplasia ossificans progressiva is a rare genetic disease characterized by widespread soft tissue ossification and congenital stigmata of the extremities.
Camargo, E E, Moura-Ribeiro, M V, Nucci, A, Queiroz, L D, Santos, A D   +4 more
core   +1 more source