Science
Explore conditions, clinical trials, and the latest research.
These pages contain non-exhaustive scientific information about uses of products that have not yet been approved by regulatory authorities, as their efficacy and safety for these uses have not yet been established. The information is provided for educational and scientific exchange purposes, and does not suggest products should be used in a manner not consistent with their respective label approved by the regulatory authorities, which may differ by geography. Any publication reflects the state of knowledge at the time it was published. The information is not medical advice and cannot replace the independent medical judgement of a healthcare professional.
ACT-EARLY
ATTRv
A clinical trial studying the prevention of variant transthyretin amyloidosis (ATTRv), also known as hereditary ATTR.
PROPEL
Achondroplasia
Our clinical program includes multiple achondroplasia clinical trials that are designed to better understand FGFR3-driven skeletal dysplasias and to evaluate an oral investigational therapy option for these conditions.
ACCEL
Hypochondroplasia
The ACCEL clinical program includes multiple studies grounded in learning more about hypochondroplasia and evaluating an oral investigational therapy option for children and adolescents with this type of skeletal dysplasia.