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July 2, 2021 1:00 pm - 1:30 pm

Opening Remarks

Chandler C. Crews Conference Host / The Chandler Project; Achondroplasia Research Conference
July 2, 2021 1:30 pm - 2:30 pm

Achondroplasia & Hypochondroplasia

This year, we are so excited to highlight Hypochondroplasia throughout the conference. Achondroplasia and hypochondroplasia are very similar, but also different from one another – Dr. Mackenzie is a world-renowned expert in both conditions.

William G. Mackenzie, MD Chairman - Department of Orthopaedics / Nemours/Alfred I. duPont Hospital for Children
July 2, 2021 2:30 pm - 3:30 pm

Vosoritide for Hypochondroplasia and other genetic conditions

Similar to achondroplasia, hypochondroplasia is also a form of short-limbed dwarfism. Dr. Andrew Dauber is the lead investigator for the study of Vosoritide for Selected Genetic Causes of Short Stature. In his presentation, Dr. Dauber will go in-depth on his study and how it benefits those with hypochondroplasia and other genetic conditions.

Andrew Dauber, MD, MMSc Chief of Endocrinology / Children’s National Hospital
July 2, 2021 3:30 pm - 4:30 pm

Achondroplasia Research & Developments

Dr. Ravi leads the largest site worldwide of international trials testing a drug on the growth of children with achondroplasia. A few of Dr. Ravi’s current projects include clinical trials of CNP for achondroplasia and skeletal dysplasia natural history studies. In his presentation, Dr. Ravi will go in-depth on what new research and developments surrounding […]

Ravi Savarirayan, MB, BS (Adel.); MD (Melb.); FRACP; ARCPA (Hon.) Group Leader of Skeletal Biology and Disease / Murdoch Children's Research Institute
July 2, 2021 5:30 pm - 6:30 pm

Cocktail Reception

Please join us for a cocktail reception at 5:30 PM on the Rooftop Terrace.

July 2, 2021 6:30 pm - 8:30 pm


For attendees on-site, dinner will follow the cocktail reception in the Brightons room.

July 3, 2021 8:00 am - 8:30 am

Breakfast + Opening Remarks

July 3, 2021 8:30 am - 8:55 am

Limb Lengthening for Dwarfism

For patients with dwarfism who opt to go through limb lengthening treatment, it’s very important they go to an institute where there is a team of experts specialized in not only limb lengthening, but dwarfism as well. In his presentation, Dr. Herzenberg will give an in-depth overview of how limb alignment and limb lengthening are […]

John E. Herzenberg, MD, FRCSC, FAAOS Director of Pediatric Orthopedics / International Center for Limb Lengthening; Rubin Institute for Advanced Orthopedics; Sinai Hospital
July 3, 2021 8:55 am - 9:20 am

Surgical Advancements + New Techniques

Limb lengthening has been available to patients with dwarfism for several decades now. Just like how pharmaceutical treatments are being developed for achondroplasia, limb lengthening treatment is constantly advancing as well – having a less invasive approach due to new techniques that are being developed daily. In his presentation, Dr. Standard will highlight what patients […]

Shawn C. Standard, MD, FAAOS Head of Pediatric Orthopedics / International Center for Limb Lengthening; Rubin Institute for Advanced Orthopedics; Sinai Hospital
July 3, 2021 9:20 am - 9:45 am

FAQs & Misconceptions on Limb Lengthening for Dwarfism

Limb lengthening is sometimes seen as a controversial procedure for those with dwarfism, mostly due to misinformation about the procedure itself. Dr. McClure is here to answer your most pressing questions about all things limb lengthening for dwarfism, as well as debunk any myths there are.

Philip K. McClure, MD Assistant Director / International Center for Limb Lengthening; Rubin Institute for Advanced Orthopedics; Sinai Hospital
July 3, 2021 9:45 am - 10:10 am

Physical Therapy + Post Lengthening Recovery

Physical therapy is a critical part of deformity correction (bowed legs) and limb lengthening treatment and recovery. At the International Center for Limb Lengthening, Anil Bhave leads a team of physical and occupational therapists specializing in treating patients with achondroplasia going through these procedures.  Rehabilitation and Physical Therapy at the International Center for Limb Lengthening

Anil Bhave, PT Clinical Director of Orthopedic Rehabilitation; Director of Wasserman Gait Laboratory / International Center for Limb Lengthening; Rubin Institute for Advanced Orthopedics; Sinai Hospital
July 3, 2021 10:20 am - 10:50 am

Vosoritide for Achondroplasia

The latest information on Vosoritide for Achondroplasia. Vosoritide is an investigational analog of C-type Natriuretic Peptide (CNP) under evaluation for the treatment of children with achondroplasia, the most common form of skeletal dysplasia.   

Laura Guido, PharmD Medical Director, Global Medical Affairs / BioMarin Pharmaceutical Inc.
July 3, 2021 10:50 am - 11:20 am

Recifercept for Achondroplasia

The latest information on Pfizer‘s pipeline for Recifercept, “a biologic medicine that targets mutated fibroblast growth factor Receptor 3 (FGFR3).” 

Christian Czech, PhD Senior Director, Head of Research / Pfizer
July 3, 2021 11:30 am - 12:00 pm

TransCon CNP for Achondroplasia

The latest information on the development of TransCon CNP for patients with achondroplasia (ACH). TransCon CNP is an investigational long-acting prodrug of CNP in development for the treatment of ACH in children.  

Ascendis Pharma, Speaker TBA Ascendis Pharma
July 3, 2021 12:00 pm - 12:30 pm

Infigratinib for Achondroplasia

The latest information on the development of Infigratinib for patients with achondroplasia. Infigratinib is an orally bioavailable and selective FGFR1/2/3 selective tyrosine kinase inhibitor in development for FGFR-related conditions. Infigratinib in children with achondroplasia (ACH): design of PROPEL2 – a phase 2, open-label, dose-escalation and dose-expansion study

Anne Lee Director, Global Patient Advocacy / QED Therapeutics
Daniela Rogoff, MD, Phd Vice President, Clinical Development / QED Therapeutics
July 3, 2021 12:30 pm - 2:00 pm

Lunch & Closing Remarks