Little legend: Child with rare form of dwarfism begins his biggest adventure yet
A child’s first day of school can be a daunting experience – both for the new student and their anxious parents. But when your child is just a little bit…
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#RareDiseaseDay 2019
Out now – new official #RareDiseaseDay video! Watch Filip and Alina’s story. He is from Romania and lives with achondroplasia Share the video to raise awareness of rare diseases for Rare Disease Day 2019. #ShowYourRare…
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The Chandler Project
As our first project, we are currently working on a weekend event dedicated to learning about new research and surgical options for achondroplasia. Our goal is to then turn this into…
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BioMarin Doses First Participant in Phase 2 Study of Vosoritide for Treatment of Infants and Young Children with Achondroplasia
SAN RAFAEL, Calif., June 14, 2018 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that the company dosed the first participant in a global Phase 2 study for vosoritide, an analog of C-type…
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Limb lengthening surgery helps kids with dwarfism reach new heights
Putting on a pair of shoes is something many of us take for granted every day. But for 7-year-old Anna Booth, those simple tasks are now big achievements. “I can…
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Therachon Announces Dosing of First Subject in Phase 1 Clinical Trial Evaluating TA-46, a Novel Investigational Therapy for the Potential Treatment of Achondroplasia
BASEL, Switzerland, February 14, 2018 – Therachon AG, a biotechnology company focused on rare genetic diseases, today announced the dosing of the first subject in its Phase 1 trial for TA-46,…
