Ontology highlight
ABSTRACT:
SUBMITTER: Palhares DB
PROVIDER: S-EPMC6409115 | biostudies-literature | 2019 Feb
REPOSITORIES: biostudies-literature
Palhares Durval Batista DB Nascimento Deborah Ribeiro DR Palhares Marilene Garcia MG Lopes Suzana S de Liane L Cristhina Paula P Mauro José J Alves Fabiana F Vieira Rancisco Oliveira RO Souza-Fagundes Elaine Maria EM Underwood Adam A Milsted Amy A Augusto Robson R Martins Almir Sousa AS
Intractable & rare diseases research 20190201 1
Fibrodysplasia ossificans progressiva (FOP) is a rare, intractable and devastating genetic connective tissue disorder characterized by progressive ectopic ossification in the soft tissues and skeleton. Three patients, one teenage girl (P1), one male adult (P2) and one male child (P3), were studied and treated with FOPCON (combined formulation of 14 mg of propranolol and 250 mg of ascorbic acid), given three times per day. P1 started treatment in March 2012, P2 in October 2012 and P3 in July 2015 ...[more]