Results 51 to 60 of about 44,644 (146)

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

Troubles oro-faciaux dans Fibrodysplasia ossificans progressiva. Revue systématique. [PDF]

, 2019
Fibrodysplasia ossificans progressiva (FOP) or myositis ossificans is a very rare autosomic dominant disease. Progressive ectopic bone formation occurs in ligaments, muscles and fascia and provoque severe movement limitation with time.
Garcia-Lopez, Carlos, Garcia, Paola, Coibion, France   +2 more
core   +3 more sources

Activin-dependent signaling in fibro/adipogenic progenitors causes fibrodysplasia ossificans progressiva

Nature Communications, 2018
Fibrodysplasia ossificans progressiva is a severe disorder characterized by heterotopic ossification, and is caused by mutations in ACVR1. Here, the authors show that expression of mutant ACVR1 in fibro/adipogenic progenitors recapitulates disease ...
John B. Lees-Shepard, Masakazu Yamamoto, Arpita A. Biswas, Sean J. Stoessel, Sarah-Anne E. Nicholas, Cathy A. Cogswell, Parvathi M. Devarakonda, Michael J. Schneider, Samantha M. Cummins, Nicholas P. Legendre, Shoko Yamamoto, Vesa Kaartinen, Jeffrey W. Hunter, David J. Goldhamer   +13 more
doaj   +1 more source

Cardiopulmonary and Neurologic Dysfunctions in Fibrodysplasia Ossificans Progressiva

Biomedicines, 2021
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, Xiaobing Yu, Edward C. Hsiao   +2 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

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

Anaesthetic considerations in a child with fibrodysplasia ossificans progressiva [PDF]

, 2019
Fibrodysplasia ossificans progressiva (FOP) is an extremely rare genetic disorder characterised by extraskeletal ossification of connective tissue. Affected individuals often become completely immobilised by their third decade of life.
Kleyenstüber, Thomas
core  

Intermittent and Short‐Term Empirical Ruxolitinib Regimen for Steroid‐Refractory Flareups of Fibrodysplasia Ossificans Progressiva

Pharmacology Research &Perspectives, Volume 13, Issue 6, December 2025.
ABSTRACT Fibrodysplasia ossificans progressiva (FOP) is an ultra‐rare genetic disorder with inflammation‐related flare‐ups resulting in catastrophic heterotopic ossification (HO). Janus‐associated kinase (JAK) inhibitors may have had a blocking effect on bone formation in controlling FOP flare‐ups by blocking multiple inflammatory signaling pathways ...
Rong‐Long Chen, Tzu‐Hsien Yang, Yun‐Hsin Wang, Jy‐juinn Shaw, Liuh‐Yow Chen   +4 more
wiley   +1 more source

Early diagnosis of fibrodysplasia ossificans progressiva

, 2008
BACKGROUND. Fibrodysplasia ossificans progressiva is a rare and disabling genetic condition characterized by congenital malformation of the great toes and by progressive heterotopic ossification in specific anatomic patterns.
Glaser, DL, Sillence, D, Shore, EM, Collins, F, Connor, M, Ravitsky, V, Zasloff, M, Xu, MQ, Zackai, E, Kitterman, J, Ganguly, A, Kaplan, FS   +11 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