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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"

ReAch Achondroplasia Registry

2016-04-11Research
Prof. Krejcis team is researching FGF signaling mechanisms, in particular, pathological FGFRs, such as FGFR3, which causes achondroplasia. His team also manages ReACH, standing for Registry of Achondroplasia, which means to serve as a tool for clinicians and healthcare providers to conduct research.

Patent and license agreement for CNP

2016-03-28Research
This news article, written by 4-traders and the pharma letters, describes the path that led to the agreement between Chugai, who discovered CNP, and BioMarin.

Science with no results shared

2016-03-04Research
Researchers are failing to submit their clinical trials' results in clinicaltrials.gov.

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.
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