Biotech Industry Liaison Committee Review

Little People of America’s (LPA) Biotech Industry Liaison Committee has established a comprehensive overview of the latest biotechnology developments in skeletal dysplasias or dwarfisms. Prior to any interaction with LPA, pharmaceutical companies began developing pharmacologic therapies. This review will focus on achondroplasia but will evolve to include developments for other conditions as they advance. The LPA Biotech Industry Liaison Committee was formed to process these ongoing research efforts to ensure the LPA community, as well as those just being introduced to dwarfism, have access to objective and easy-to-understand information in which to base healthcare decisions. In this review, we break down the latest data and identify questions that still need to be answered.  (Click here to download a PDF version of this review)

LPA’s Position Statement

Little People of America (LPA), the world’s oldest and largest support organization for people with dwarfism and their families, has been tracking biotechnical developments to treat individuals with Achondroplasia. With the FDA approval of Biomarin’s Voxzogo therapy, the dwarfism community is challenged by the impact of this new treatment on our health, culture, and our standing within society.

LPA strongly believes that a pharmaceutical intervention focusing on growth velocity is not addressing the actual health and quality of life issues of people with dwarfism. Our height-related challenges are primarily based on inaccessible architecture, lack of universal design, and society’s intolerance and discrimination of people with short stature. We are concerned that this recently approved drug attempts a pharmaceutical solution to a societal issue. Our community acknowledges that the hardships associated with having dwarfism need to be addressed. Yet, we strive for our height to be reframed as a part of the diversity of humanity. We want to reprioritize research goals to be the most meaningful ones for our members, such as reducing spinal stenosis, sleep apnea, and the need for corrective surgeries.

Our role is to provide social support and advocacy for our members and their community. As part of this role, LPA supports medical research, especially that which improves the quality of life of our members by treating symptoms that can range from uncomfortable to lethal. We respect the personal choices of all families or individuals regarding healthcare decisions and welcome all individuals and families to be part of LPA, regardless of medical decisions and outcomes. As medical science moves forward, we will continue to inform researchers about the value of dwarf pride and its contributions to human biological, social, and cultural diversity.

What is Achondroplasia:
Medical management of achondroplasia:
Research overview: 
Current development:

About LPA’s Biotech Industry Liaison Committee:

This committee is a diverse, well-rounded group of LPA members that was formed to provide objective, unbiased interpretation, and analysis of the ongoing research efforts in skeletal dysplasias. Our committee includes both the short-statured and average height perspective including lifetime LPA members, new parents, as well as caregivers to children who have participated in some of the clinical trials. Together, we bring a robust knowledge in healthcare, pharmaceutical industry, legal, and executive board experience.

Committee Members:

  • Michael Hughes, Chair
  • Dianna Carda
  • Sue Chohan
  • Colleen Gioffreda
  • Ashley Grist
  • Deb Himsel
  • Kelsie Hankins Hughes
  • Nancy Kaplan
  • Michelle Kraus
  • Josh Maudlin
  • Karen McDonnell
  • Michael Petruzzelli
  • Mark Povinelli

Contact email: [email protected]