Ipsen Receives Complete Response Letter for Palovarotene, an Investigational Treatment for Fibrodysplasia Ossificans Progressiva
The CRL is related to the U.S. FDA’s previous request for additional information on palovarotene clinical trial data Ipsen anticipates responding to the request in the first quarter of 2023 PARIS, FRANCE, 23 December 2022– The... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - December 23, 2022 Category: Drugs & Pharmacology Source Type: news

Ipsen Announces U.S. FDA Priority Review for Palovarotene New Drug Application in Patients With Fibrodysplasia Ossificans Progressiva Following Resubmission
PARIS--(BUSINESS WIRE)--Regulatory News: June 29, 2022 -- Ipsen (Euronext: IPN; ADR: IPSEY) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review its resubmitted New Drug Application (NDA) for... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - June 29, 2022 Category: Drugs & Pharmacology Source Type: news

Ipsen Confirms U.S. FDA Accepts New Drug Application for Palovarotene as the First Potential Treatment Worldwide for Fibrodysplasia Ossificans Progressiva (FOP)
PARIS, FRANCE, 28 May 2021– Ipsen (Euronext: IPN; ADR: IPSEY) today announced that its New Drug Application (NDA) for palovarotene, an oral, investigational, selective RARγ agonist for the prevention of heterotopic ossification... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - May 28, 2021 Category: Drugs & Pharmacology Source Type: news

Augmented BMP signaling commits cranial neural crest cells to a chondrogenic fate by suppressing autophagic {beta}-catenin degradation
Cranial neural crest cells (CNCCs) are a population of multipotent stem cells that give rise to craniofacial bone and cartilage during development. Bone morphogenetic protein (BMP) signaling and autophagy have been individually implicated in stem cell homeostasis. Mutations that cause constitutive activation of the BMP type I receptor ACVR1 cause the congenital disorder fibrodysplasia ossificans progressiva (FOP), which is characterized by ectopic cartilage and bone in connective tissues in the trunk and sometimes includes ectopic craniofacial bones. Here, we showed that enhanced BMP signaling through the constitutively ac...
Source: Signal Transduction Knowledge Environment - January 12, 2021 Category: Science Authors: Yang, J., Kitami, M., Pan, H., Nakamura, M. T., Zhang, H., Liu, F., Zhu, L., Komatsu, Y., Mishina, Y. Tags: STKE Research Articles Source Type: news

Woman, 35, is 'turning into a statue' due to a one-in-a-million condition
Rachel Winnard, 35, of Rochdale, Greater Manchester, suffers from the genetic disorder Fibrodysplasia Ossificans Progressiva, which causes bony bars to form within the body's muscles. (Source: the Mail online | Health)
Source: the Mail online | Health - August 7, 2019 Category: Consumer Health News Source Type: news

The woman who is slowly 'turning to STONE': Condition causes muscle to gradually change into bone 
Carli Henrotay, of Saint Louis, Missouri, has the genetic condition fibrodysplasia ossificans progressiva. With a life expectancy of 40, it gets progressively worse with age and has no cure. (Source: the Mail online | Health)
Source: the Mail online | Health - May 28, 2019 Category: Consumer Health News Source Type: news

Hope for people with disease that turns tissue to BONE as three drugs enter clinical trial
Three drugs have entered clinical trials as potential therapies for those with fibrodysplasia ossificans progressiva, a genetic condition that turns dasmaged tissue and muscle into bone. (Source: the Mail online | Health)
Source: the Mail online | Health - March 20, 2019 Category: Consumer Health News Source Type: news

Heartbreaking stories of patients battling a rare condition 'that is slowly turning them into stone'
Thozi Mciki, 50, and Damian Fredericks, 18, both from near Cape Town in South Africa, have a rare condition called fibrodysplasia ossificans progressiva which causes muscles to turn to bone. (Source: the Mail online | Health)
Source: the Mail online | Health - October 26, 2018 Category: Consumer Health News Source Type: news

Ballymena sisters have a disease turning them to stone
Zoe Buxton and Lucy Fretwell, 26, from Ballymena, Northern Ireland, have a one-in-two-million condition, called fibrodysplasia ossificans progressiva, which causes bone to replace muscle. (Source: the Mail online | Health)
Source: the Mail online | Health - July 17, 2017 Category: Consumer Health News Source Type: news

Sisters, 26, have a disease that is turning them to STONE
Zoe Buxton and Lucy Fretwell, 26, from Ballymena, Northern Ireland, have a one-in-two-million condition, called fibrodysplasia ossificans progressiva, which causes bone to replace muscle. (Source: the Mail online | Health)
Source: the Mail online | Health - July 17, 2017 Category: Consumer Health News Source Type: news

Rare condition causes BONE to form in woman's muscles
Jasmin Floyd, from Connecticut, suffers with Fibrodysplasia Ossificans Progressiva - a rare muscle disorder which has only 800 recorded cases worldwide. (Source: the Mail online | Health)
Source: the Mail online | Health - March 16, 2017 Category: Consumer Health News Source Type: news

Wigan man turned stiff from ultra rare condition FOP dies
Adrian Berry, 40, from Wigan, suffered from fibrodysplasia ossificans progressiva. He passed away with pneumonia on Boxing Day (pictured with friends on his 40th birthday). (Source: the Mail online | Health)
Source: the Mail online | Health - January 19, 2017 Category: Consumer Health News Source Type: news

Her body is slowly growing a second skeleton
It's impossible for those who have fibrodysplasia ossificans progressiva, or FOP, to describe what it's like to live with it. (Source: CNN.com - Health)
Source: CNN.com - Health - December 26, 2016 Category: Consumer Health News Source Type: news

Quieting cells' low-oxygen alarm stops flare-ups in rare bone disorder
The cellular response to the lack of oxygen fans the flames of flare-ups in a rare bone disorder. In fibrodysplasia ossificans progressiva, a mutation triggers bone growth in muscles, which limits motion, breathing, and swallowing, among a host of progressive symptoms. The study identifies a therapeutic target for stopping the extra bone growth in FOP and other disorders. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - May 2, 2016 Category: Science Source Type: news

Drug candidate stops extra bone growth in animal model of rare, genetic disease
New preclinical research provides support to a drug that has been repurposed to possibly treat a rare and extremely disabling genetic bone disease, particularly in children. In that disease, fibrodysplasia ossificans progressiva (FOP), a mutation triggers bone growth in muscles, alters skeletal bone formation, and limits motion, breathing, and swallowing, among a host of progressive symptoms. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - April 13, 2016 Category: Science Source Type: news