Results 71 to 80 of about 7,648 (236)

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

How Activin A Became a Therapeutic Target in Fibrodysplasia Ossificans Progressiva

Biomolecules
Fibrodysplasia ossificans progressiva (FOP) is a rare genetic disorder characterized by episodic yet cumulative heterotopic ossification (HO) of skeletal muscles, tendons, ligaments, and fascia. FOP arises from missense mutations in Activin Receptor type
Dushyanth Srinivasan, Martin Arostegui, Erich J. Goebel, Kaitlin N. Hart, Senem Aykul, John B. Lees-Shepard, Vincent Idone, Sarah J. Hatsell, Aris N. Economides   +8 more
doaj   +1 more source

ACVR1/JAK1/JAK2 inhibitor momelotinib reverses transfusion dependency and suppresses hepcidin in myelofibrosis phase 2 trial.

Blood Advances, 2020
Momelotinib (MMB) is a JAK1/2 and ACVR1 inhibitor with demonstrated clinical activity in all 3 hallmarks of myelofibrosis (MF): anemia, constitutional symptoms, and splenomegaly.
S. Oh, M. Talpaz, A. Gerds, Vikas Gupta, S. Verstovsek, R. Mesa, Carole B. Miller, C. Rivera, Angela G. Fleischman, S. Goel, M. Heaney, C. O'Connell, M. Arcasoy, Yafeng Zhang, J. Kawashima, T. Ganz, M. Kowalski, C. Brachmann   +17 more
semanticscholar   +1 more source

Recent Topics in Fibrodysplasia Ossificans Progressiva [PDF]

Endocrinology and Metabolism, 2018
Fibrodysplasia ossificans progressiva (FOP) is a rare genetic disease that is characterized by the formation of heterotopic bone tissues in soft tissues, such as skeletal muscle, ligament, and tendon.
Takenobu Katagiri, Sho Tsukamoto, Yutaka Nakachi, Mai Kuratani   +3 more
doaj   +1 more source

Differential involvement of Wnt signaling in Bmp regulation of cancellous versus periosteal bone growth [PDF]

, 2017
Bone morphogenetic proteins (Bmp) are well-known to induce bone formation following chondrogenesis, but the direct role of Bmp signaling in the osteoblast lineage is not completely understood.
Bellido, Teresita, He, Guangxu, Lim, Joohyun, Long, Fanxin, Ni, Jiangdong, Shi, Yu   +5 more
core   +3 more sources

An Acvr1 R206H knock-in mouse has fibrodysplasia ossificans progressiva [PDF]

Journal of Bone and Mineral Research, 2012
Abstract Fibrodysplasia ossificans progressiva (FOP; MIM #135100) is a debilitating genetic disorder of dysregulated cellular differentiation characterized by malformation of the great toes during embryonic skeletal development and by progressive heterotopic endochondral ossification postnatally.
Salin A, Chakkalakal, Deyu, Zhang, Andria L, Culbert, Michael R, Convente, Robert J, Caron, Alexander C, Wright, Andrew D A, Maidment, Frederick S, Kaplan, Eileen M, Shore   +8 more
openaire   +2 more sources

Fibrodysplasia Ossificans Progressiva: A rare case series

Journal of Orthopaedic Reports, 2023
Background: Fibrodysplasia ossificans progressiva is a rare autosomal dominant connective tissue disorder with a prevalence of 2 per million individuals.
Lokesh kumar Sekaran, Nirosha Ponnuraj, Vijayaraja Elangovan, Dheepan Kumar Sakthimohan   +3 more
doaj   +1 more source

The TGFβ type I receptor TGFβRI functions as an inhibitor of BMP signaling in cartilage. [PDF]

, 2019
The type I TGFβ receptor TGFβRI (encoded by Tgfbr1) was ablated in cartilage. The resulting Tgfbr1 Col2 mice exhibited lethal chondrodysplasia. Similar defects were not seen in mice lacking the type II TGFβ receptor or SMADs 2 and 3, the intracellular ...
Baek, Jongseung, Chun, Hyelim, Lopez, Noah, Lyons, Karen M, Razavi, Peyman, Sadik, Joshua Elyahu, Shirazyan, Anna, Wang, Weiguang   +7 more
core  

Sjögren’s syndrome-associated microRNAs in CD14+ monocytes unveils targeted TGFβ signaling [PDF]

, 2016
SjS-associated miRNA expression levels are positively associated in CD14+ monocytes. Linear regression analyses were used to define associations between miRNAs in HC (circle, n = 17), SLE (triangle, n = 17), and RA (diamond, n = 18) patient groups ...
Adrienne E. G. Williams, Annie L. Chan, Carol M. Stewart, Kevin Choi, Michael R. Bubb, Seunghee Cha, Westley H. Reeves, Yun Jong Lee   +7 more
core   +4 more sources

Repurposing Vandetanib plus Everolimus for the Treatment of ACVR1-Mutant Diffuse Intrinsic Pontine Glioma

Cancer Discovery, 2021
Combined vandetanib and everolimus was identified by artificial intelligence as a potential therapy for ACVR1-mutant DIPG, as they synergize in vitro and extend survival in vivo, with preliminary testing in four children suggesting this combination as a ...
Diana M. Carvalho, Peter J. Richardson, N. Olaciregui, Reda Stankunaite, C. Lavarino, V. Molinari, Elizabeth A Corley, Daniel P. Smith, R. Ruddle, Adam Donovan, A. Pál, F. Raynaud, Sara Temelso, A. Mackay, J. Overington, A. Phelan, David Sheppard, Ande . A. MAcKINNON, B. Zebian, S. Al-Sarraj, A. Merve, J. Pryce, J. Grill, M. Hubank, O. Cruz, A. Morales La Madrid, S. Mueller, A. Carcaboso, F. Carceller, Chris Jones   +29 more
semanticscholar   +1 more source