Results 61 to 70 of about 3,838 (202)
Superior mesenteric artery syndrome in a patient with fibrodysplasia ossificans progressiva
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 +3 more
doaj +1 more source
Fibrodysplasia Ossificans Progressiva: Case Report. [PDF]
Fibrodysplasia ossificans progressiva is a rare genetic disease characterized by widespread soft tissue ossification and congenital stigmata of the extremities.
Camargo, E E +4 more
core +1 more source
The Scientific Case for Animal Models: A Perspective From Musculoskeletal Researchers
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 +4 more
wiley +1 more source
گزارش 1 مورد بيمار مبتلا به فيبروديسپلازی(ميوزيت) اسيفيکان پيشرونده [PDF]
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
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 +9 more
wiley +1 more source
International physician survey on management of FOP: a modified Delphi study
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 +12 more
doaj +1 more source
Fibrodysplasia Ossificans Progressiva: Literature Review and Case Report
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 +3 more
doaj +1 more source
Heterotopic Ossification: Cellular Basis, Symptoms, and Treatment [PDF]
Heterotopic ossification (HO) is the process by which calcified bone develops in soft tissues. Because of the abnormal calcification, complications such as bone deformation, loss of range of motion, and joint immobility adversely affect patients.
Wolfe, Brian
core
Surrogate Endpoints in Regulatory Decision‐Making
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
Cardiopulmonary and Neurologic Dysfunctions in Fibrodysplasia Ossificans Progressiva
Fibrodysplasia Ossificans Progressiva (FOP) is an ultra-rare but debilitating disorder characterized by spontaneous, progressive, and irreversible heterotopic ossifications (HO) at extraskeletal sites.
Fatima Khan +2 more
doaj +1 more source

