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15th April 2020

Ascendis Pharma shared some notes with patient organizations advocating for achondroplasia to inform patients and families on the progression of the observational study, ACHieve, and interventional study, ACcomplisH, for achondroplasia during the COVID-19 pandemic.

Most sites that have started the observational study ACHieve, will lift restrictions only when all the current pandemic situation is overcome to ensure the safety of children and access to investigator site staff for future monitoring visits.

Ascendis professionals remain hopeful that children can start receiving TransCon CNP as soon as possible. The company continues to monitor and adapt to the impact of COVID-19 and to work towards escalating sequential dose cohorts throughout the year 2020 while ensuring the safety of the children during the current pandemic and also ensuring there is the availability of the clinical centers' staff for future monitoring visits.

About TransCon CNP

TransCon CNP is an investigational long-acting prodrug of C-type natriuretic peptide (CNP) in development as a therapy for children with achondroplasia (ACH), the most common form of dwarfism, for which there is no FDA-approved treatment. TransCon CNP is designed to provide continuous exposure of CNP at safe, therapeutic levels via a single, weekly subcutaneous dose: -- The ACcomplisH Trial is a global, phase 2, randomized, double-blind, placebo-controlled trial designed to evaluate the safety and efficacy of TransCon CNP at escalating doses in children (ages 2 to 10 years) with ACH. 


A brief explanation of some words and concepts

Sites- clinical centers where the studies are taking place

Escalating sequential dose cohorts - groups of children receiving different doses of TransCon CNP

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