4th Annual Achondroplasia Research Conference

Last week, The Chandler Project (TCP) hosted the 4th Annual Achondroplasia Research Conference in Chicago, IL. Although this was the fourth conference, it was only the third one where we could get together in person – our biggest in-person attendance yet! Last year, the conference introduced research in hypochondroplasia as a focus. This year, in addition to achondroplasia AND hypochondroplasia research, we introduced research surrounding Spondyloepiphyseal Dysplasia Congenita (SEDc). From here on out, TCP is excited to expand coverage on research surrounding other forms of dwarfism and skeletal dysplasia. Who knows what next year may bring?

This year, conference-goers heard from lead investigators, world-renowned physicians, and 5 pharmaceutical companies over a span of two days. Presentations included:

Diagnostic and Therapeutic Approach in the Growth Hormone Era
Dr. Andrew Dauber, Chief of Endocrinology
Children’s National Hospital

First in Class Transformative Therapies for Patients with Rare Bone Disorders
Samantha Parker, Chief Patient Access Officer
InnoSkel

New Precision Therapies for FGFR3-Related Skeletal Dysplasia
Professor Ravi Savarirayan MB, BS (Adel.); MD (Melb.); FRACP; ARCPA (Hon.)Group Leader of Skeletal Therapies
Murdoch Children’s Research Institute

CLARITY: Achondroplasia Natural History Study
Dr. Jeffrey Campbell
Pediatric Neurosurgery
Nemours Children’s Hospital

The Spine in Achondroplasia: Why Treat? Why Fuse?
Dr. David Feldman
Associate Director
Paley Orthopedic & Spine Institute at St. Mary’s Medical Center

Orthopedic Care for Achondroplasia
Dr. Philip McClure
Assistant Director
International Center for Limb Lengthening, Rubin Institute for Advanced Orthopedics at Sinai Hospital

Limb Lengthening for Achondroplasia
Dr. Shawn Standard
Head of Pediatric Orthopedics
International Center for Limb Lengthening, Rubin Institute for Advanced Orthopedics at Sinai Hospital

Development of Recifercept: Potential for Use in Achondroplasia
Alison SladeGCL & Clinician
Rare Disease Research Unit, Pfizer

Targeting FGFR in Achondroplasia and Other FGFR-Driven Skeletal Dysplasia
Carl Dambkowski, MD
Chief Medical Officer, QED Therapeutics

Ascendis Pharma’s Commitment to Achondroplasia
Adebola Giwa, MD
Associate Medical Director of Clinical Development, Ascendis Pharma

Panel Q&A
Facilitated by Dr. Cathleen RaggioCo-Director
The Katrhyn O. and Alan C. Greenberg Center for Skeletal DysplasiasHospital for Special Surgery

VOXZOGO® (vosoritide) for Injection: One Family’s Story (presentation not recorded)
Janet Legare, MD
Clinical Director
Waisman Center at University of Wisconsin-Madison + BioMarin Clinical Coordinator and a parent of a child receiving VOXZOGO®

If you attended the conference (or registered by June 18) you can access the recorded sessions by logging into the conference site with the account you created when you registered. If you did not attend the conference, but want to view the recordings, you can register as a conference attendee (fee applies)