Go behind the scenes of this year’s Drug Development Forum When Dan Perrien attended his first FOP Drug Development Forum (DDF), he met two young children with fibrodysplasia ossificans progressiva (FOP). He watched them run and play like any other children would — while the adults in the room were on high alert to make […]
Meet 12-year-old Raina When Raina and her friends created a club called “The Underpuppies,” they decided on just one requirement: You have to be genuinely kind to join. To enforce that requirement, they developed a test. The current Underpuppies stage a situation where the prospective member has to help someone in need or be willing […]
The Fibrodysplasia Ossificans Progressiva Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the […]
Purpose of the study Fibrodysplasia ossificans progressiva (FOP) is a rare autosomal dominant condition that leads to significant disability and morbidity, characterised by the formation of heterotopic hard tissues within connective tissues. The condition has an incidence of approximately one per two million people worldwide. There is no known single effective treatment available for FOP. […]
Abstract Introduction and importance Fibrodysplasia Ossificans Progressiva is an ultra-rare genetic disorder of progressive soft tissue ossification. Due to unawareness and poor clinical suspicion, the rate of misdiagnosis, delay in diagnosis, and unnecessary diagnostic procedures leading to permanent injury and lifelong disability is common. Here we report this rare genetic disorder in a six years […]
Introduction Fibrodysplasia ossificans progressiva (FOP) is a rare, progressively debilitating disorder first described in 1692 by Guy Patin [1]. This disease is characterized by progressive heterotopic ossification that forms normal bone in characteristic extraskeletal sites and congenital malformations of the great toes. Progressive episodes of heterotopic ossification occur in characteristic pattern first at the dorsal, axial, […]
Action The U.S. Food and Drug Administration (FDA) has approved Sohonos (palovarotene) capsules for reduction in the volume of new heterotopic ossification (extra-skeletal bone formation) in adults and children aged 8 years and older for females, and 10 years and older for males with fibrodysplasia ossificans progressiva. Sohonos is the first drug approved for patients with fibrodysplasia […]
A multisite, international phase 2 trial evaluating the investigational drug garetosmab has shown that it reduced soft-tissue flare-ups significantly and prevented new areas of abnormal bone formation in patients with fibrodysplasia ossificans progressiva (FOP). Kathryn Dahir, MD, professor of Medicine in the Division of Endocrinology and Diabetes, served as the principal investigator at Vanderbilt University […]
