Cargando…

Prevalence of fibrodysplasia ossificans progressiva (FOP) in the United States: estimate from three treatment centers and a patient organization

BACKGROUND: Fibrodysplasia ossificans progressiva (FOP), an ultra-rare, progressive, and permanently disabling disorder of extraskeletal ossification, is characterized by episodic and painful flare-ups and irreversible heterotopic ossification in muscles, tendons, and ligaments. Prevalence estimates...

Descripción completa

Detalles Bibliográficos
Autores principales:	Pignolo, Robert J., Hsiao, Edward C., Baujat, Genevieve, Lapidus, David, Sherman, Adam, Kaplan, Frederick S.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2021
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8340531/ https://www.ncbi.nlm.nih.gov/pubmed/34353327 http://dx.doi.org/10.1186/s13023-021-01983-2

Ejemplares similares

Fibrodysplasia Ossificans Progressiva (FOP): A Segmental Progeroid Syndrome
por: Pignolo, Robert J., et al.
Publicado: (2020)

Special considerations for clinical trials in fibrodysplasia ossificans progressiva (FOP)
por: Hsiao, Edward C., et al.
Publicado: (2018)

Palovarotene for Fibrodysplasia Ossificans Progressiva (FOP): Results of a Randomized, Placebo‐Controlled, Double‐Blind Phase 2 Trial
por: Pignolo, Robert J., et al.
Publicado: (2022)

OR29-05 A Natural History Study of Fibrodysplasia Ossificans Progressiva (FOP): 12-Month Outcomes
por: Mukaddam, Mona Al, et al.
Publicado: (2020)

Prevalence of fibrodysplasia ossificans progressiva (FOP) in France: an estimate based on a record linkage of two national databases
por: Baujat, Geneviève, et al.
Publicado: (2017)

Fibrodysplasia Ossificans Progressiva: Clinical and Genetic Aspects
por: Pignolo, Robert J, et al.
Publicado: (2011)

Current challenges and opportunities in the care of patients with fibrodysplasia ossificans progressiva (FOP): an international, multi-stakeholder perspective
por: Pignolo, Robert J., et al.
Publicado: (2022)

PSAT180 Fibrodysplasia Ossificans Progressiva (FOP) A Case Series
por: Almonaei, Khulod, et al.
Publicado: (2022)

Compartment Syndrome of the Thigh in a Patient with Fibrodysplasia Ossificans Progressiva
por: Kaplan, Frederick S., et al.
Publicado: (2020)

Study methodology and insights from the palovarotene clinical development program in fibrodysplasia ossificans progressiva
por: Pignolo, Robert J., et al.
Publicado: (2023)

SUN-344 Patients with Fibrodysplasia Ossificans Progressiva Have an Increased Prevalence of Cardiac Conduction Abnormalities
por: Kou, Samuel, et al.
Publicado: (2020)

Natural history of fibrodysplasia ossificans progressiva: cross-sectional analysis of annotated baseline phenotypes
por: Pignolo, Robert J., et al.
Publicado: (2019)

Clinical, evolution and therapeutical considerations upon a case of fibrodysplasia ossificans progressiva (FOP)
por: Rogoveanu, O, et al.
Publicado: (2013)

Fibrodysplasia ossificans progressiva
por: Smit, Chané, et al.
Publicado: (2023)

Correction to: Natural history of fibrodysplasia ossificans progressiva: cross-sectional analysis of annotated baseline phenotypes
por: Pignolo, Robert J., et al.
Publicado: (2019)

Anti-ACVR1 antibodies exacerbate heterotopic ossification in fibrodysplasia ossificans progressiva (FOP) by activating FOP-mutant ACVR1
por: Aykul, Senem, et al.
Publicado: (2022)

Crohn's Disease in a Patient With Fibrodysplasia Ossificans Progressiva
por: Havlichek, Daniel, et al.
Publicado: (2022)

Cardiopulmonary and Neurologic Dysfunctions in Fibrodysplasia Ossificans Progressiva
por: Khan, Fatima, et al.
Publicado: (2021)

Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles
por: Eekhoff, Elisabeth M.W., et al.
Publicado: (2022)

The Developmental Phenotype of the Great Toe in Fibrodysplasia Ossificans Progressiva
por: Towler, O. Will, et al.
Publicado: (2020)

Application of in vitro Drug Metabolism Studies in Chemical Structure Optimization for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP)
por: Padilha, Elias C., et al.
Publicado: (2019)

Fibrodysplasia ossificans progressiva: mechanisms and models of skeletal metamorphosis
por: Kaplan, Frederick S., et al.
Publicado: (2012)

Longitudinal Evaluation of Pain, Flare‐Up, and Emotional Health in Fibrodysplasia Ossificans Progressiva: Analyses of the International FOP Registry
por: Peng, Ke, et al.
Publicado: (2019)

Recent Topics in Fibrodysplasia Ossificans Progressiva
por: Katagiri, Takenobu, et al.
Publicado: (2018)

Quality of life of patients with fibrodysplasia ossificans progressiva
por: Ortiz-Agapito, Fernando, et al.
Publicado: (2015)

Bone Resection Osteotomy in Fibrodysplasia Ossificans Progressiva
por: Colmenares-Bonilla, Douglas, et al.
Publicado: (2018)

Fibrodysplasia Ossificans Progressiva: A Challenging Diagnosis
por: De Brasi, Daniele, et al.
Publicado: (2021)

Fibrodysplasia Ossificans Progressiva and Pregnancy: A Case Series and Review of the Literature
por: Forrest, Alexandra D., et al.
Publicado: (2022)

Suppression of heterotopic ossification in fibrodysplasia ossificans progressiva using AAV gene delivery
por: Yang, Yeon-Suk, et al.
Publicado: (2022)

Limb specific Acvr1‐knockout during embryogenesis in mice exhibits great toe malformation as seen in Fibrodysplasia Ossificans Progressiva (FOP)
por: Hildebrand, Laura, et al.
Publicado: (2019)

“I Can’t Take off My Shirt or Do My Own Hair”—A Qualitative Investigation of the Symptoms and Impact Experience of Children and Adolescents with Fibrodysplasia Ossificans Progressiva (FOP)
por: Markowitz, Jessica T., et al.
Publicado: (2022)

The Diversity of the Clinical Phenotypes in Patients With Fibrodysplasia Ossificans Progressiva
por: Al Kaissi, Ali, et al.
Publicado: (2016)

Challenges and Opportunities for Drug Repositioning in Fibrodysplasia Ossificans Progressiva
por: Ventura, Francesc, et al.
Publicado: (2021)

Monitoring and Management of Fibrodysplasia Ossificans Progressiva: Current Perspectives
por: Smilde, Bernard J, et al.
Publicado: (2022)

Fibrodysplasia Ossificans Progressiva: A Report of Four Cases
por: Elamin, Mohamed, et al.
Publicado: (2022)

Saracatinib is an efficacious clinical candidate for fibrodysplasia ossificans progressiva
por: Williams, Eleanor, et al.
Publicado: (2021)

Recent progress in drug development for fibrodysplasia ossificans progressiva
por: Meng, Xinmiao, et al.
Publicado: (2022)

Social and Clinical Impact of COVID-19 on Patients with Fibrodysplasia Ossificans Progressiva
por: Kou, Samuel, et al.
Publicado: (2021)

Social and clinical impact of COVID-19 on patients with fibrodysplasia ossificans progressiva
por: Kou, Samuel, et al.
Publicado: (2022)

Whole-body Computed Tomography Versus Dual Energy X‑ray Absorptiometry for Assessing Heterotopic Ossification in Fibrodysplasia Ossificans Progressiva
por: Warner, Sarah E., et al.
Publicado: (2021)

Cannot write session to /tmp/vufind_sessions/sess_a28cv4237pisjgh15r75kt6047