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FDA Accepts TransCon® CNP NDA for Priority Review
SOURCE: Investors.AscendisPharma.com (June 2, 2025) – Once-weekly TransCon CNP demonstrated significantly higher annualized growth velocity, the primary endpoint, compared to placebo – Multiple benefits beyond linear growth were observed compared to placebo, with a safety and tolerability profile similar to placebo – The FDA grants Priority Review designation for the evaluation of medicines that, if approved, would provide a significant improvement in the safety or effectiveness of…
latest neWs
pathways to navigating achondroplasia
my achon journey
The Chandler Project partnered with QED Therapeutics to develop a new educational resource designed to support individuals and families living with achondroplasia. MyAchonJourney provides reliable information and practical tools from pregnancy through the first five years of a child’s life, empowering families on their journey.
researching achondroplasia & other skeletal dysplasias
the following companies have or are developing pipelines for achondroplasia and other skeletal dysplasias
Biomarin, Qed Therapeutics, Ascendis Pharma, and tyra biosciences have sponsored & or supported events and initiatives led by the chandler project.
achondroplasia & skeletal dysplasia RARE-X data collection program
By generating the most comprehensive data collection program, you can help accelerate research and the development of new drugs, devices, or other therapies for achondroplasia and other skeletal dysplasias.
ACHONDROPLASIA RESEARCH
drug may reduce sudden infant death syndrome in children with achondroplasia
Vosoritide therapy in children with achondroplasia aged 3-59 months: a multinational, randomized, double-blind, placebo-controlled, phase 2 trial
Correspondence to: Professor Ravi Savarirayan, Murdoch Children’s Research Institute, Royal Children’s Hospital, University of Melbourne, Parkville, VIC 3054, Australia
ravi.savarirayan@vcgs.org.au
2024 ACHONDROPLASIA & SKELETAL DYSPLASIA RESEARCH CONFERENCE
The 6th Annual Achondroplasia & Skeletal Dysplasia Research Conference brought together patients, parents and caregivers, physicians, researchers, and pharmaceutical and biotech companies to engage and network with one another in a safe environment to openly discuss the latest research and developments surrounding pharmaceutical treatment options and advances in surgical treatment for achondroplasia and other skeletal dysplasias.
The Chandler Project
Through advocacy, The Chandler Project raises awareness of transformative research on achondroplasia and other skeletal dysplasias by supporting a global community and network of patients, parents, and caregivers seeking information on the latest pharmaceutical research & developments and advancements in surgical treatment.
The Chandler Project is mission-oriented to bringing parents & caregivers of newly diagnosed babies science from the source by connecting them with world-renowned researchers and physician experts who treat achondroplasia and other skeletal dysplasias. Click here to learn more
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FDA Accepts TransCon® CNP NDA for Priority Review
SOURCE: Investors.AscendisPharma.com (June 2, 2025) – Once-weekly TransCon CNP demonstrated significantly higher annualized growth velocity, the primary endpoint, compared to placebo – Multiple benefits beyond linear growth were observed compared to placebo, with a safety and tolerability profile similar to placebo – The FDA grants Priority Review designation for the evaluation of medicines that, if approved, would provide a significant…
