4:00-6:00 PM

A/V test for presenters

6:00-8:00 PM

Registration + Family Meet & Greet

9:00-9:30 AM

Patient to PharmD

Sivani Gadiraju, PharmD

9:30-10:00 AM

Limb Alignment + Limb Lengthening for Achondroplasia

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

*This presentation will be streamed, Dr. Standard is unable to attend in person this year.

10:00-10:30 AM

The Development of Recifercept for Achondroplasia

Michael P. Wajnrajch, MD, MPA
Senior Medical Director, Rare Endocrine & IEM Team Lead, Pfizer

Clinical Professor of Pediatrics, Division of Pediatric Endocrinology, NYU Langone Medical Center

10:30-10:40 AM

Break

10:40-11:15 AM

PROPEL Program: Targeting FGFR in Achondroplasia

Daniela Rogoff, MD, PhD
Senior VP, Clinical Development

QED therapeutics, a BridgeBio company

11:15-12:15 PM

Keynote Presentation

Dr. Ravi Savarirayan
Group Leader for Molecular Therapies
Murdoch Children's Research Institute

12:15-1:00 PM

Lunch

1:00-1:30 PM

Pediatric Approach to Achondroplasia

Dr. Janet Legare
Pediatrician of Clinical Genetics
University of Wisconsin School of Medicine and Public Health

1:30-2:30 PM

One Family’s Story: A Guide to Starting VOXZOGO (vosoritide)

Hear from an expert physician and a mom of a child taking Voxzogo. Event is for families with a child age 5 and older.

Philippe Backeljauw, MD
Pediatric Endocrinologist Cincinnati Children’s Hospital Medical Center

Tracey R.
Caregiver and mom to Nora

2:30-2:40 PM

Break

2:30-3:00 PM

Achondroplasia and Areas of Unmet Needs

Anglea Hu, MD
Associate Clinical Research Director, Rare Blood and Bone Disorders

Sanofi S. A

3:00-3:30 PM

TYRA-300, an FGFR3 Specific Inhibitor

Ronald V. Swanson
Chief Scientific Officer

Tyra Biosciences 

3:30-4:00 PM

Introduction to Ascendis Pharma and TransCon Technology

John Caminis
U.S. Head of Medical Strategy & Engagement

Ascendis Pharma

5:30-6:30 PM

Q&A Panel moderated by Dr. Janet Legare

Panel Q&A with Pfizer, Sanofi, Tyra Biosciences, Ascendis Pharma, QED therapeutics, and Dr. Ravi Savarirayan

6:30-8:30 PM

Dinner

10:00AM-12:00 PM

Rising Love: A Children’s Book about bread baking and achondroplasia

Have fun baking with us and learn about Rising Love. Children are welcome! Event is open to families and children of all ages.

Dr. Janet Legare