Results 41 to 50 of about 44,644 (146)

Analysis of Real‐World Data Utilization in the Orphan Drug Approval Process: Focusing on New Drug Marketing Applications Submitted to the FDA

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

Anesthetic and airway management for inguinal hernia repair in a pediatric patient with fibrodysplasia ossificans progressiva: a case report

Journal of Medical Case Reports
Background Fibrodysplasia ossificans progressiva is a rare genetic disorder characterized by the progressive heterotopic ossification of soft connective tissues, ultimately leading to cumulative disability and ankylosis.
Saori Ozaki, Satoshi Kamiya, Noriko Takeno, Tomomi Ishii, Yukari Toyota, Hiroshi Yokomi, Takahiro Kato, Yasuo M. Tsutsumi   +7 more
doaj   +1 more source

An mTOR Signaling Modulator Suppressed Heterotopic Ossification of Fibrodysplasia Ossificans Progressiva

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, Chengzhu Zhao, Kazuhiko Horigome, Megumi Nishio, Yasue Okanishi, Sanae Nagata, Shingo Komura, Yasuhiro Yamada, Junya Toguchida, Akira Ohta, Makoto Ikeya   +10 more
doaj   +1 more source

Supply and Demand in the Mathematics of Rare Disease Drug Development: Why Choosing the Right Model Is Crucial

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

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

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, Joseph J. Abbo, Matthew J. Yousif   +2 more
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

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

Evaluation of clinical cardiac safety of zilurgisertib, an activin receptor‐like kinase‐2 (ALK2) inhibitor, in healthy participants

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, Hong Yang, Xing Liu, Xiaohua Gong, Jay Getsy, Kevin Rockich   +5 more
wiley   +1 more source

Catastrophic Falls in Patients Who Have Fibrodysplasia Ossificans Progressiva [PDF]

, 1998
There have been numerous anecdotal reports of catastrophic falls in patents with fibrodysplasia ossificans progressiva. To determine the incidence of serious morbidity and mortality associated with falls in this patent population, the authors surveyed ...
Glaser, David, Rocke, David, Kaplan, Frederick S.   +2 more
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