Results 11 to 20 of about 1,727 (105)

Intermittent and Short-Term Empirical Ruxolitinib Regimen for Steroid-Refractory Flareups of Fibrodysplasia Ossificans Progressiva. [PDF]

Pharmacol Res Perspect
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 ...
Chen RL, Yang TH, Wang YH, Shaw JJ, Chen LY.   +4 more
europepmc   +2 more sources

Unexpected Promotion of Bone Regeneration by Inhibition of BMPR1A‐Mediated BMP Signalling

Cell Proliferation, EarlyView.
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, Yun Zhai, Xiaochen Liu, Jiaojiao Shao, Jiani Zhou, Zhaoyang Li, Jiale He, Shuxian Lin, Qi Zhang   +8 more
wiley   +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, Yunlan Fang, Phillip Wang, Xiang Liu, Jay Getsy, Kevin Rockich   +5 more
wiley   +1 more source

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

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

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

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

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

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