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Industry Programme

Saturday 27 June 2026,  13:00-14:00

A satellite symposium organised by BridgeBio

Evolving Standards of Care in Achondroplasia: A Lifespan Approach from Prenatal Diagnosis to Adulthood

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SYNOPSIS:

Achondroplasia is associated with evolving medical needs across the lifespan that benefit from coordinated, multidisciplinary care and inclusive, person-centered approaches.

This industrysponsored symposium will provide a comprehensive, lifespan perspective on care for individuals with achondroplasia from prenatal recognition and early infancy through adolescence and adulthood, highlighting collaborative care models, recent therapeutic advances, and ongoing gaps and opportunities to improve the standard of care for individuals with achondroplasia.

FACULTY:

Janet Legare, MD
Professor of Pediatrics, University of Wisconsin School of Medicine and Public Health Director Midwest Regional Bone Dysplasia Clinic

Svein Fredwall, MD, PhD
Sr. Consultant and Medical Advisor TRS National Resource Centre for Rare Disorders, Sunnaas Rehabilitation Hospital, Nesodden, Norway

Saturday 27 June 2026,  17:45-18:45

A satellite symposium organised by BioMarin

Solving the hypochondroplasia diagnostic puzzle:
Exploring key pieces through case examples

OBJECTIVES:

Interactive discussion of clinical cases to highlight key diagnostic challenges in hypochondroplasia, and to identify practical strategies to improve and accelerate diagnosis through earlier recognition of red-flag symptoms and optimized diagnostic approaches.

PROGRAMME:

Welcome and introductions
Philippe Campeau

Exploring key pieces of the hypochondroplasia diagnostic puzzle through clinical cases: 

Unmet needs: A caregiver’s experience
Reni Pekala

Recognizing clinical red flags 
Julie Hoover-Fong

Strategies in genetic testing
Peter Kannu

Solving the hypochondroplasia diagnostic puzzle: Key takeaways
Philippe Campeau

Q&A 
All speakers

FACULTY:

Philippe Campeau (Medical geneticist)
CHU Sainte-Justine Research Center, Montreal, QC, Canada

Reni Pekala (PAG representative)
Hypochondroplasia Families Community, Greystones, Ireland

Julie Hoover-Fong (Medical geneticist)
Johns Hopkins University School of Medicine, Baltimore, MD, USA

Peter Kannu (Medical geneticist)
University of Alberta-Stollery Children’s Hospital, Edmonton, Canada

Sunday 28 June 2026,  17:30-19:00

A satellite symposium organised by Alexion

The Hypophosphatasia (HPP) Disease Continuum From Children to Adults: Early Diagnosis and Effective Management

PROGRAMME:

Welcome and introductions
Dr Eric T. Rush

Pediatric Perspectives: Diagnosing and Managing HPP
Dr Oliver Semler

Transition of Care: Disease Burden and Management in Adolescents and Adults
Dr Eric T. Rush

Pioneering New Possibilities for HPP Care
Dr Raja Padidela

Q&A Session
All

Alexion medicines in the context of HPP management will be discussed at this meeting. Some content may be deemed promotional in your territory.This satellite symposium is fully funded and organized by Alexion, AstraZeneca Rare Disease, and is intended for healthcare professionals only.

FACULTY:

Dr Eric T. Rush – Chair
Children’s Mercy Hospital, Kansas City, MO, USA, University of Missouri, Kansas City, Kansas City, MO, USA

Dr Oliver Semler
Children’s Hospital, University Cologne, Cologne, Germany

Dr Raja Padidela
Royal Manchester Children’s Hospital, Manchester, UK
The University of Manchester, Manchester, UK

Monday 29 June 2026,  15:45-16:45

A satellite symposium organised by Kyowa Kirin

Rare Matters in XLH
From Infancy to Adolescence and Beyond

PROGRAMME:

Welcome and introduction to XLH
Chelsey Grimbly

Are we looking for the obvious in early XLH diagnosis?
Anya Rothenbuhler

Minimizing morbidity in XLH
Thomas Carpenter

Isn’t XLH just a childhood disease?
Jill Simmons

Key takeaways and closing remarks 
Chelsey Grimbly

FACULTY:

Chelsey Grimbly – Chair (Canada) 

Thomas O. Carpenter (USA)

Jill Simmons (USA)

Anya Rothenbuhler (France)

Novo Nordisk is a leading global healthcare company, founded in 1923 and headquartered in Denmark. Our Rare Disease division is focused on generating scientific and technological breakthroughs for people living with a rare disease via the discovery and development of integrated therapeutic solutions and novel indications of established medicines in rare and ultra-rare blood, endocrine and renal disorders. Novo Nordisk employs about 50,800 people in 80 countries out of which 3.300 within rare disease.

WEBSITE

Kyowa Kirin strives to create and deliver novel medicines with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company with a heritage of 70+ years, we apply cutting-edge science including an expertise in antibody research and engineering, to address the needs of patients and society across multiple therapeutic areas including Nephrology, Oncology, Immunology/Allergy and Neurology. Across four regions – Japan, Asia Pacific, North America and EMEA/International – we focus on our purpose, to make people smile, and are united by our shared values of commitment to life, teamwork/Wa*, innovation and integrity.

*Harmony and loop among people.

WEBSITE

Founded in 2007, Ascendis Pharma is applying its innovative TransCon™ technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by our core values of patients, science and passion, we use our TransCon technologies to fulfill our mission of developing new and potentially best-in-class therapies that address unmet medical needs. Ascendis is headquartered in Copenhagen, Denmark, and has additional facilities in Europe and the United States.

WEBSITE

Inozyme Pharma is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases driven by pathologic mineralization and intimal proliferation which impact the vasculature, soft tissue and skeleton. We are initially focused on developing an enzyme therapy to treat ENPP1 and ABCC6 Deficiencies, rare genetic diseases associated with significant morbidity, mortality, and unmet medical need.

WEBSITE

BioMarin is a world leader in developing and commercializing innovative therapies for rare diseases driven by genetic causes. With a 20-year history, BioMarin remains steadfast to its original mission—to bring new treatments to market that will make a big impact on small patient populations. These conditions are often inherited, difficult to diagnose, progressively debilitating, have few, if any, treatment options, and are usually ignored. Visit www.biomarin.com to learn more.

WEBSITE

Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, Inc. As a leader in rare diseases for 30 years, Alexion is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialization of life-changing medicines. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on hematology, nephrology, neurology, metabolic disorders, cardiology and ophthalmology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries.

WEBSITE