About the Conference

Bringing together achondroplasia patients and their caregivers to meet and hear from world-renowned physicians and researchers who are leading experts in the treatment of achondroplasia. This conference is a hybrid event. The health and safety of patients and conference attendees are our top priority. Due to COVID-19, this will be a hybrid event, thus offering attendees the […]

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Why should you attend?

The ONLY event that brings together leading pharmaceutical companies and some of the top world-renowned orthopedic experts specialized in treating achondroplasia.

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2
Days
8
Institutes & Companies
14
Speakers
Chandler C. Crews
Conference Host The Chandler Project; Achondroplasia Research Conference
William G. Mackenzie, MD
Chairman - Department of Orthopaedics Nemours/Alfred I. duPont Hospital for Children
Andrew Dauber, MD, MMSc
Chief of Endocrinology Children’s National Hospital
Ravi Savarirayan, MB, BS (Adel.); MD (Melb.); FRACP; ARCPA (Hon.)
Group Leader of Skeletal Biology and Disease Murdoch Children's Research Institute
John E. Herzenberg, MD, FRCSC, FAAOS
Director of Pediatric Orthopedics International Center for Limb Lengthening; Rubin Institute for Advanced Orthopedics; Sinai Hospital
Shawn C. Standard, MD, FAAOS
Head of Pediatric Orthopedics International Center for Limb Lengthening; Rubin Institute for Advanced Orthopedics; Sinai Hospital
Philip K. McClure, MD
Assistant Director International Center for Limb Lengthening; Rubin Institute for Advanced Orthopedics; Sinai Hospital
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
Laura Guido, PharmD
Medical Director, Global Medical Affairs BioMarin Pharmaceutical Inc.
Christian Czech, PhD
Senior Director, Head of Research Pfizer
Carl Decker, PhD
Clinical Scientist, Clinical Development Ascendis Pharma
Adebola Giwa, MD
Associate Medical Director, Clinical Development Ascendis Pharma
Daniela Rogoff, MD, Phd
Vice President, Clinical Development QED Therapeutics
Anne Lee
Director, Global Patient Advocacy QED Therapeutics

Agenda

1:00 pm - 1:30 pm

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

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
2:30 pm - 3:30 pm

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
3:30 pm - 4:30 pm

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
5:30 pm - 6:30 pm

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

6:30 pm - 8:30 pm

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

8:00 am - 8:30 am

8:30 am - 8:55 am

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
8:55 am - 9:20 am

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
9:20 am - 9:45 am

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
9:45 am - 10:10 am

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
10:20 am - 10:50 am

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.
10:50 am - 11:20 am

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
11:30 am - 12:00 pm

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.  

Adebola Giwa, MD Associate Medical Director, Clinical Development
Ascendis Pharma
Carl Decker, PhD Clinical Scientist, Clinical Development
Ascendis Pharma
12:00 pm - 12:30 pm

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
12:30 pm - 2:00 pm

Venue

Royal Sonesta Harbor Court Baltimore

550 Light St. Baltimore, MD 21202

Located on the picturesque waterfront, the Royal Sonesta Harbor Court Baltimore brings refinement, charm and laid-back style that stands-out among Baltimore, Maryland hotels.

Blog

Achondroplasia

Growing Strong

Bay Area parents Munira and Amer Haider talk about the fund they established to help support research on Achondroplasia,... Read More
Achondroplasia
BioMarin

Novel therapy improves height for children with achondroplasia

via Healio/Endocrine Today Children with a common form of skeletal dysplasia saw persistent and sustained improvements in annualized growth... Read More
Achondroplasia

Achondroplasia Growth Charts

For growth monitoring of patients with achondroplasia by Lo Neumeyer, Andrea Merker, Lars Hagenäs Clinical surveillance of infants and children... Read More