Fibrodysplasia Ossificans Progressiva News and Research

RSS

Optimized genome-editing method opens the door to more effective treatment of genetic diseases

CRISPR-Cas9 is widely used to edit the genome by studying genes of interest and modifying disease-associated genes.

11 Apr 2023

Discovery opens up the possibility of pursuing new therapies for FOP

Fibrodysplasia ossificans progressiva (FOP) is a rare disease characterized by extensive bone growth outside of the normal skeleton that pre-empts the body's normal responses to even minor injuries. It results in what some term a "second skeleton," which locks up joint movement and could make it hard to breathe.

16 Jan 2022

Research may lead to first-ever pharmacological treatment for rare genetic bone disease

Scientists at Children's Hospital of Philadelphia report that a drug candidate that blocks abnormal protein signals may lead to the first pharmacologic treatment for hereditary multiple exostoses (HME), a rare pediatric genetic disease.

29 Aug 2017

Researchers examine critical role of hypoxia in induction, amplification of FOP lesions

The cellular response to the lack of oxygen fans the flames of flare-ups in a rare bone disorder. In fibrodysplasia ossificans progressiva (FOP), a mutation triggers bone growth in muscles, which limits motion, breathing, and swallowing, among a host of progressive symptoms.

3 May 2016

Palovarotene drug may prevent multiple musculoskeletal problems linked with FOP

New research in laboratory animals suggests that the drug palovarotene may prevent multiple skeletal problems caused by a rare but extremely disabling genetic bone disease, and may even be a candidate for use in newborn babies with the condition.

15 Apr 2016

Clementia expands enrollment to include children with FOP in ongoing Phase 2 clinical trial

Clementia Pharmaceuticals, Inc. today announced the initiation of enrollment of children as young as 6 years old in the company's ongoing Phase 2 study of palovarotene for the treatment of fibrodysplasia ossificans progressiva (FOP).

26 Aug 2015

Clementia Pharmaceuticals commences multi-center study of patients with fibrodysplasia ossificans progressiva

Clementia Pharmaceuticals, Inc. today announced that it has commenced enrollment in the second part (Part B) of its natural history study in patients with fibrodysplasia ossificans progressiva (FOP), a rare, severely disabling congenital myopathy characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) that result in the formation of new, abnormal (heterotopic) bone in muscles, tendons and ligaments.

28 May 2015

Clementia's palovarotene receives FDA Fast Track designation for FOP treatment

Clementia Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration has granted Fast Track designation to palovarotene, which is being developed for the treatment of fibrodysplasia ossificans progressiva (FOP).

1 Dec 2014

Clementia gets Orphan Medicinal Product Designation from EMA for palovarotene

Clementia Pharmaceuticals, Inc. announced today that the European Medicines Agency (EMA) has granted Orphan Medicinal Product Designation for palovarotene, the company's lead product candidate, for the treatment of fibrodysplasia ossificans progressiva (FOP).

21 Nov 2014

Clementia initiates Phase 2 extension study of palovarotene in FOP patients

Clementia Pharmaceuticals, Inc. announced today the initiation of a Phase 2 extension study of palovarotene in patients with fibrodysplasia ossificans progressiva (FOP), a rare, severely disabling genetic disease characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) and new abnormal bone formation.

27 Oct 2014

Genome Project offers new leads to improve outcomes for children with high-grade glioma brain tumors

The St. Jude Children's Research Hospital-Washington University Pediatric Cancer Genome Project has identified new mutations in pediatric brain tumors known as high-grade gliomas (HGGs), which most often occur in the youngest patients.

8 Apr 2014

Researchers use stem cells to study bone disorders from patients afflicted with genetic bone disease

Researchers have developed a new way to study bone disorders and bone growth, using stem cells from patients afflicted with a rare, genetic bone disease.

9 Jan 2014

Researchers to begin drug development projects for rare and neglected diseases

Researchers will begin drug development projects for rare and neglected diseases that include potential treatments for a musculoskeletal disorder, a cognitive dysfunction disorder, a virus that affects the central nervous system of newborns, a parasitic worm infection, a form of muscular dystrophy and a rare lung disease.

16 Nov 2011

New genetic approach to treat fibrodysplasia ossificans progressiva

Scientists at Penn's Perelman School of Medicine Center for Research in FOP and Related Disorders have developed a new genetic approach to specifically block the damaged copy of the gene for a rare bone disease, while leaving the normal copy untouched.

9 Nov 2011

Research: Substance P appears to trigger formation of extraskeletal bone

For hundreds of thousands of people, injuring a muscle through an accident like falling off a bike or having surgery can result in a strange and serious complication. Their muscles start growing bones.

21 Jul 2011

New research reveals potential treatment for HO in children, soldiers

Promising new research reveals a potentially highly effective treatment for heterotopic ossification, a painful and often debilitating abnormal buildup of bone tissue. HO comes in two main forms—one that appears in children and is congenital, another that strikes wounded military personnel and surgery patients and is triggered by severe injuries and wounds.

4 Apr 2011

Scientists to develop a treatment for rare genetic disorder, FOP

An international team of scientists, led by researchers at the University of Pennsylvania School of Medicine, is taking the first step in developing a treatment for a rare genetic disorder called fibrodysplasia ossificans progressiva (FOP), in which the body's skeletal muscles and soft connective tissue turns to bone, immobilizing patients over a lifetime with a second skeleton.

13 Nov 2009

Penn researchers discover gene that creates "second skeleton" bone disorder

Researchers at the University of Pennsylvania School of Medicine have located the "skeleton key," a gene that, when damaged, causes the body's skeletal muscles and soft connective tissue to undergo a metamorphosis into bone, progressively locking joints in place and rendering movement impossible.

23 Apr 2006

Only 10 percent of doctors have ever heard of fibrodysplasia ossificans progressiva

A new research study shows how common a medical misdiagnosis can be and how severely it can exacerbate a disease.

7 Nov 2005