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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"

Speech Therapy

Speech therapy is a form of therapy that works to evaluate, prevent, intervene and study communication disturbances (speech, written and non-verbal) and swallowing disorders. Anyone who aims to treat these types of disorders can benefit from speech therapy, from newborns to the elderly, improving their quality of life.

Research on ARQ 087, a Tyrosine kinase inhibitor

News on the Tyrosine Kinase Inhibitors (TKIs). A new paper was published by Dr. Krejci and his team, suggesting, for the second time, that TKIs are unsuitable to treat achondroplasia, but can still be useful for treating various forms of cancer.

[Free Full Text] Final adult height in long-term growth hormone-treated achondroplasia patients

There are several therapies that increase height in achondroplasia patients. Among these, growth hormone (GH) administration has been a controversial subject for not contributing significantly to final height in patients who underwent this treatment.

[Free Full Text] RBM 007 - new approach for achondroplasia

8 projects were named for pre-designation review as orphan drug commercialization candidates, which includes a potential drug for achondroplasia, RMB-007, an aptamer being developed by Ribomic. Learn more about it here.

New inhibitor of FGFR3 in mouse model

Controversial data has been published about tyrosine kinase inhibitors (TKIs) as drugs for the treatment of achondroplasia. The first paper shows that in cell-free assays these compounds have shown to be effective as a treatment, but toxic in newborn mice. The second paper ([Free Full Text]) suggests that one of these compounds has shown to be effective as treatments of thanatophoric dysplasia.

Roadmap for a new therapy - by BioMarin

This infographic summarizes achondroplasia in a very simple way and shows the steps that are needed to find new therapies for this disease.
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