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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"

Limb Lengthening And Deformity Correction Surgery

2017-11-29Current approaches
The innovative treatments in development for achondroplasia are focused on the increase of growth rate and in the reduction of possible complications. The only available treatment is surgical and is known as limb lengthening surgery. For those who need to correct a deformity, such as genu varus, deformity correction surgery is a necessity, and during this procedure, height may or may not be increased at the same time, depending on the patient choice and physician opinion. Limb lengthening can he […]
Ribomic logo for RMB-007

RMB-007

2017-11-28Emergent treatments
RMB-007 is a potential drug being developed by Ribomic Inc., a Japanese biotech company. It is still in development and is still in pre-clinical studies.

Meclozine

2017-11-22Emergent treatments
Meclozine (or Meclizine) is an over-the-counter drug for motion sickness that has been shown to be a potential treatment for achondroplasia by Prof. Matsushita. It has already been tested in mice and dogs and is heading for Phase I.

TransCon CNP

2017-11-16Emergent treatments
TransCon CNP is a new molecule for achondroplasia under development by Ascendis Pharma. Its mechanism of action is similar to the one used by Vosoritide, but with a modification in a formulation that makes a big difference. Learn more about it here.
Therachon's TA-46

TA-46 Mechanism Of Action

2017-11-16Emergent treatments
TA-46 is a soluble form of FGFR3. This receptor that exists in chondrocytes is where is the mutation in achondroplasia is located. Phase 1 clinical trials for this molecule will start in early 2018. Learn more about how it works and how far it is in development here.
Biomarin

BMN-111 Mechanism of Action

2017-06-15Emergent treatments
BMN-111 is an emergent treatment for achondroplasia. It is now in a Phase III clinical trial and aims to bring a treatment to the condition. In this article you can read more on how it works, its strong suits and drawbacks.
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