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Survey about achondroplasia
We have designed a questionnaire with the purpose to better know the population connected to achondroplasia.
The questionnaire is optional and anonymous. The data obtained will be used for statistical purposes and to better understand the natural history of achondroplasia. Please consider answering.
If you would like to take the survey later, you can click on the link at the top "Register at BA"

Phase 3 trial for achondroplasia - BioMarin Study 111-301

Highlights of the Phase 3 clinical trial design for BMN-111, developed by BioMarin. Learn more about how it's going to be processed here.

The TransCon CNP, a prodrug for achondroplasia

Ascendis Pharma is developing a drug for achondroplasia, using new technology that combines the benefits of a pro-drug and sustained-release technologies. Learn more about TransCon CNP here.

A challenging report on Vosoritide

Although BioMarin's results for Vosoritide have been mostly positive, there are some concerns regarding the less positive results.

BioMarin presents report on phase 2 study of Vosoritide

BioMarin has issued a new press release with data on the phase 2 clinical trial for Vosoritide, during the ASHG 2016 meeting. Read all about it here.

State of the art in achondroplasia

This is a summary of the current state of drug development in achondroplasia.

BMN-111 Phase 2: study 111-202 and extension study 111-205

There are two studies by BioMarin in Phase 2 clinical trials where BMN-111 (vosoritide) is being administered. They have different objectives and durations, which can be consulted here.
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