(Pathology) a skeletal disorder, characterized by failure of normal conversion of cartilage into bone, that begins during fetal life and results in dwarfism
Ascendis Pharma announced the filing of an investigational new drug, or IND, application with the FDA to initiate the ACcomplisH Trial, a global phase 2 trial designed to evaluate the safety and efficacy of TransCon CNP in children with achondroplasia. TransCon CNP is a long-acting prodrug of C-type natriuretic peptide, or CNP, in development for children with achondroplasia.
Release date- 18062019 - Researchers at Johns Hopkins Medicine, the Murdoch Children's Research Institute in Australia and seven other medical institutions report that an experimental drug called vosoritide, which interferes with certain proteins that block bone growth, allowed the average annual growth rate to increase in a study of 35 children and teenagers with achondroplasia, a form of dwarfism.
United States-based Pfizer has signed a definitive agreement to acquire all the shares of Therachon Holding AG, a privately-held clinical-stage biotechnology company focused on rare diseases, with assets in development for the treatment of achondroplasia and short bowel syndrome, it was reported yesterday.
Under the terms of the agreement, Pfizer will acquire Therachon for USD 340m upfront with an additional USD 470m in additional payments contingent on the achievement of key milestones in the development and commercialisation of TA-46 for the treatment of achondroplasia, a genetic condition and the most common form of short-limbed dwarfism.
The literature data on the use of growth hormone in skeletal dysplasias are scarce except for in achondroplasia, and usually short-term treatment results have been reported (9).
(7) Infantile Tay-Sachs disease, for example, uncontroversially reduces well-being, while many have argued that deafness and achondroplasia (the most common type of dwarfism) do not reduce well-being at all.
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