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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"

Achondroplasia Natural History Multicenter Clinical Study

2016-02-21Research
This study not only means to understand the natural history of achondroplasia (what complications occur, how often, average life span, etc.), but also aims to build a global patient registry. What is the purpose of a patient registry? Know more about it here.

Vosoritide Proceeding to Phase 3

2016-02-02Research
What are the next steps for BMN-111? Here you can find a timetable for what is planned for this rdug in development.

To better understand CRISPR-Cas9

2016-01-03Research
New treatments using the CRISPR/Cas9 gene editing tool are being developed. In order to understand how these would work here is a short vídeo and article about this possible excision of the mutation and substitution with the non-mutated gene.

Cutting Off The Mutation

2016-01-01Research
CRISPR/Cas9 is powerful new gene editing tool, making development of new treatments easier, faster and cheaper. It's potential for use in humans is currently under study, but how does it work exactly, and why is it so versatile? Learn more about this revolutionary technology here.

BioMarin investors page announces

2015-11-25Research
BioMarin intends to move into Phase 3 study discussions on the next stage of development with health authorities with a dose of 15 micrograms per kilogram daily. Also, a new cohort, designed to test a new dose regimen has completed enrollment for the Phase 2 study.

New cohort revealed for phase 2 of BMN-111

2015-10-19Research
A new cohort for the phase 2 clinical trial for BMN-111 has been revealed, learn more about it here.
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