Results 51 to 60 of about 3,838 (202)
Clinical and Translational Science, Volume 19, Issue 5, May 2026.ABSTRACT Clinical trials for rare diseases face a fundamental mathematical challenge that conventional randomized controlled trial (RCT) designs cannot overcome. With approximately 95% of the estimated 10,000–16,000 rare diseases lacking approved therapies, and drug development programs failing at rates exceeding 75% in non‐oncology indications, the ...
Marshall L. Summar, Janet Woodcock
wiley +1 more source
eLife, 2019 We respond to concerns expressed by Pacifici and Shore (2019) about a recent paper (Lees-Shepard and Goldhamer, 2018a) in which we reported that the drug palovarotene can have severe side effects in a mouse model of fibrodysplasia ossificans progressiva.
David J Goldhamer, John B Lees-Shepard
doaj +1 more source
Revision Distal Bicep Repair in the Setting of Heterotopic Ossification: Case Study
Clinical Case Reports, Volume 14, Issue 2, February 2026.Radiographic and intraoperative findings demonstrating heterotopic ossification following distal biceps tendon repair. ABSTRACT Heterotopic ossification (HO) following distal biceps tendon repair is an uncommon but potential complication. This may present pain, limited range of motion, or sensory changes due to nerve compression.
Aidan M. Brikho +2 more
wiley +1 more source
Fibrodysplasia Ossificans Progressiva - An Uncommon Cause of Trismus
Indian Journal of Physical Medicine & Rehabilitation, 2022 Fibrodysplasia ossificans progressiva (FOP) is a progressively disabling genetic disorder. It is characterised by development of a secondary skeleton due to progressive hypertrophic ossification.
Ravi Sankaran +2 more
doaj +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
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 +5 more
wiley +1 more source
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 +10 more
doaj +1 more source
Early stage fibrodysplasia ossificans progressiva: A case report
Radiology Case Reports, 2020 Fibrodysplasia ossificans progressiva is a very rare autosomal dominant genetic connective tissue disease with a progressive ectopic ossification of muscle (intramuscular) or perimuscular connective tissue such as tendons or joint capsules.
Achmad Fauzi Kamal, MD,PhD +1 more
doaj +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
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