Results 31 to 40 of about 2,003 (162)
The Turkish Journal of Pediatrics, 2018 Fibrodysplasia ossificans progressiva is one of the most devastating disorder of mankind characterized by progressive heterotopic ossification. Apart from hallux valgus, other symptoms start to develop in the first decade of life.
Mustafa Çakan +3 more
doaj +1 more source
Fibrodysplasia ossificans progressiva in Hong Kong—A case report series
Frontiers in Pediatrics, 2023 Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare condition. The diagnosis could be challenging due to its rarity and non-specific presenting symptoms.
Joshua Chun Ki Chan +10 more
doaj +1 more source
Regenerative Therapy, 2019 The demand for development of new drugs remains on the upward trend because of the large number of patients suffering from intractable diseases for which effective treatment has not been established yet.
Taiki Nakajima, Makoto Ikeya
doaj +1 more source
Unexpected Promotion of Bone Regeneration by Inhibition of BMPR1A‐Mediated BMP Signalling
Cell Proliferation, Volume 59, Issue 8, August 2026.BMPR1A downregulation reduces ID1, releases TCF3 to activate GNG4‐PI3K‐AKT signalling, thereby promoting OPC proliferation and bone regeneration. ABSTRACT Bone morphogenetic protein (BMP) signalling plays a pivotal role in bone regeneration by regulating osteoprogenitor cell (OPC) function, and BMPs have been widely used in clinical treatment. However,
Zihao Zhou +8 more
wiley +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 +3 more
doaj +1 more source
Effects of Renal and Hepatic Impairment on the Pharmacokinetics of Zilurgisertib
British Journal of Clinical Pharmacology, Volume 92, Issue 5, Page 1339-1351, May 2026.Aim Small‐molecule activin receptor‐like kinase‐2 inhibitor zilurgisertib (INCB000928) is under investigation in a pivotal trial for the treatment of fibrodysplasia ossificans progressiva (FOP), an ultrarare genetic condition. This analysis assessed effects of renal and hepatic impairment on zilurgisertib pharmacokinetics.
Yan‐ou Yang +5 more
wiley +1 more source
Clinical Pharmacology &Therapeutics, Volume 119, Issue 5, Page 1340-1349, May 2026.In the field of rare diseases—where traditional clinical trials are often impractical—real‐world data (RWD) have emerged as a scientifically valid alternative to support regulatory decision making. This study systematically evaluates the utilization of RWD in orphan drug approvals by the FDA Center for Drug Evaluation and Research (CDER) over the past ...
Minji Kim, Eunjin Hong
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
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
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