Drug trial for achondroplasia

December 14, 2012 @ No Comments

Recently, the Murdoch Children’s Research Institute in Melbourne has taken steps to beginning trials of a drug developed by the American pharmaceutical company Biomarin. The trials will be aimed at children with Achondroplasia between the ages of 4-9. The research suggest that the drug may help to reduce some of the complications associated with Achondroplasia; the need for shunts, spinal and hip surgeries etc.

Link to article in ‘The Australian’ newspaper dated 27th November 2012:


Link to article in ‘The Herald-Sun’ newspaper dated 27th November 2012:



Statement in response by the SSPA: 

“The SSPA support all of the work done by Professor Ravi Savarirayan and the team at the Murdoch Children’s Research Institute to help improve the quality of life for individuals born with the most common form of dwarfism, Achondroplasia. The new drug trial being implemented by US pharmaceutical company, Biomarin, has the potential to have a positive impact on children living with Achondroplasia by decreasing the prevalence of some of the complications associated with the condition. As the National support group for short statured people and their families, the SSPA want to ensure that families are receiving all the relevant medical, social and emotional support during the trial. We have individuals living with Achondroplasia as well as their parents available to discuss the trial with any families seeking support.”

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