Results 61 to 70 of about 3,826 (203)

First‐In‐Human Study to Assess the Pharmacokinetics and Safety of DS‐6016a After Single Subcutaneous Injection in Healthy Japanese Adults

Clinical Pharmacology in Drug Development, Volume 15, Issue 1, January 2026.
Abstract Fibrodysplasia ossificans progressiva is a rare, progressive autosomal dominant genetic disease caused by an activin receptor‐like kinase 2 (ALK2) mutation with a need for effective prophylactic therapies. This single‐center, randomized, double‐blind, placebo‐controlled study evaluated the pharmacokinetics and safety of DS‐6016a, a novel ...
Kei Okita, Hidetoshi Furuie, Akifumi Kurata, Yasuko Owada, Satoshi Yoshiba, Kei Furihata, Takaaki Oka, Yushi Kashihara, Hitoshi Ishizuka, Kazutaka Yoshihara   +9 more
wiley   +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

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

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

International physician survey on management of FOP: a modified Delphi study

Orphanet Journal of Rare Diseases, 2017
Fibrodysplasia ossificans progressiva (FOP), a disabling disorder of progressive heterotopic ossification (HEO), is caused by heterozygous gain-of- function mutations in Activin receptor A, type I (ACVR1, also known as ALK2), a bone morphogenetic protein
Maja Di Rocco, Genevieve Baujat, Marta Bertamino, Matthew Brown, Carmen L. De Cunto, Patricia L. R. Delai, Elisabeth M. W. Eekhoff, Nobuhiko Haga, Edward Hsiao, Richard Keen, Rolf Morhart, Robert J. Pignolo, Frederick S. Kaplan   +12 more
doaj   +1 more source

Miositis osificante progresiva: ultraestructura, bioquímica e histoquímica de músculo macroscópicamente sano [PDF]

, 1995
Se estudió un caso de miositis osificante progresiva en una niña de 13 años, a la cual se le tomó una muestra de músculo gastronecmio lateral, aparentemente no afectado, en el curso de una intervención quirúrgica ortopédica.
Finol, Héctor J., Garmendia, J., Hernández, N., Martínez Conde, A., Torres, S.H.   +4 more
core  

ACVR1 function in health and disease [PDF]

, 2020
Activin A receptor type I (ACVR1) encodes for a bone morphogenetic protein type I receptor of the TGFβ receptor superfamily. It is involved in a wide variety of biological processes, including bone, heart, cartilage, nervous, and reproductive system ...
Pimenta-Lopes, Carolina, Rosa López, José Luis, Sánchez de Diego, Cristina, Valer, José Antonio, Ventura Pujol, Francesc   +4 more
core   +1 more source

Developing Treatments for Fibrodysplasia Ossificans Progressiva:From preclinical concepts to practical applications [PDF]

, 2023
This thesis aimed to build upon and expand the pathophysiological and clinical knowledge of FOP and to contribute to the translation of this knowledge into new treatment strategies for FOP.
Smilde, Bernard Jan
core   +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