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Hereditary multiple exostoses: are there new plausible treatment strategies?


ABSTRACT: Introduction:Hereditary multiple exostoses (HME) is a rare congenital pediatric disorder characterized by osteochondromas forming next to the growth plates in young patients. The osteochondromas cause multiple health problems that include skeletal deformities and chronic pain. Surgery is used to remove the most symptomatic osteochondromas but because of their large number, many are left in place, causing life-long problems and increasing the probability of malignant transformation. There is no other treatment to prevent or reduce osteochondromas formation at present. Areas covered:Recent studies reviewable through PubMed are providing new insights into cellular and molecular mechanisms of osteochondroma development. The resulting data are suggesting rational and plausible new therapeutic strategies for osteochondroma prevention some of which are being tested in HME animal models and one of which is part of a just announced clinical trial. Expert Commentary:This section summarizes and evaluates such strategies and points also to possible future alternatives.

SUBMITTER: Pacifici M 

PROVIDER: S-EPMC6707746 | biostudies-literature | 2018

REPOSITORIES: biostudies-literature

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Hereditary multiple exostoses: are there new plausible treatment strategies?

Pacifici Maurizio M  

Expert opinion on orphan drugs 20180607 6


<h4>Introduction</h4>Hereditary multiple exostoses (HME) is a rare congenital pediatric disorder characterized by osteochondromas forming next to the growth plates in young patients. The osteochondromas cause multiple health problems that include skeletal deformities and chronic pain. Surgery is used to remove the most symptomatic osteochondromas but because of their large number, many are left in place, causing life-long problems and increasing the probability of malignant transformation. There  ...[more]

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