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 a bigger event where new research and medical advancements are presented for other forms of skeletal dysplasias, including achondroplasia, as well as (or and) congenital …
Pfizer Acquires Clinical-Stage Biotech Therachon
Expands Pfizer’s rare disease portfolio with potential first-in-class therapy for achondroplasia, a genetic condition and the most common form of short-limb dwarfism NEW YORK & BASEL, Switzerland–Pfizer (NYSE: PFE) today announced that it has entered into a definitive agreement to acquire all the shares of Therachon Holding AG, a privately-held clinical-stage biotechnology company focused on …
Ascendis Pharma Announces Orphan Drug Designation Granted for TransCon CNP as Treatment for Achondroplasia
COPENHAGEN, Denmark, Feb. 28, 2019 (GLOBE NEWSWIRE) — Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon™ technology to address significant unmet medical needs, today announced that the U.S. Food and Drug Administration(FDA) has granted Orphan Drug Designation (ODD) to TransCon CNP, a long-acting prodrug of C-type natriuretic peptide (CNP) in development for children with achondroplasia. Achondroplasia …
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 different – like five-year-old Floyd Henry Morley – the first day of school brings with it a whole new host of challenges. Floyd was born …
#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 Rare disease day is on February 28!