Biotech Industry Liaison Committee Review

Conclusion

We recognize that this is a sensitive and difficult topic for many individuals with dwarfism. As part of our mission to support the community of people with dwarfism, LPA will continue to advocate for researchers to be mindful of our commitment to the value of dwarf pride and its contributions to human biological, social, and cultural diversity. Furthermore, we will continue to strongly push for research efforts to focus beyond growth velocity, but instead on the outcomes most meaningful to our community, such as reducing spinal stenosis, sleep apnea, corrective surgeries, and/or improving quality of life. Currently, vosoritide is the first drug to meet its primary endpoint for achondroplasia, however, we do not know yet whether this improvement in growth velocity will translate to improving more meaningful outcomes.