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Saturday 22 June 2025

SATURDAY 11:45-12:45

A Satellite Symposium organised by BioMarin

From infancy to adolescence: reshaping achondroplasia management with vosoritide▼

PROGRAMME

Welcome & introduction – Melita Irving (London, UK)

Updates on vosoritide: long-term clinical data across ages and treatment in young children – Melita Irving (London, UK)

Real-world case studies: vosoritide in children of different ages – Heike Hoyer-Kuhn (Cologne, Germany) & Anna Allegri (Genoa, Italy)

Q&A and closing remarks – All speakers

▼ This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions. Healthcare professionals should report adverse events in accordance with their local requirements. Adverse events should also be reported to BioMarin on + 1 415 506 6179 or drugsafety@bmrn.com.
Vosoritide is indicated for the treatment of achondroplasia in patients 4 months of age and older whose epiphyses are not closed.

SATURDAY 13:45-14:45

A Satellite Symposium organised by Inozyme

Recognizing ENPP1 Deficiency An Overlooked Cause of Hypophosphatemic Rickets

PROGRAMME

Welcome and introductions – Agnès Linglart (Paris, France)

The Natural History of ENPP1 Deficiency – Generalized Arterial Calcification of Infancy and Autosomal Recessive Hypophosphatemic Rickets – David Weber (Philadelphia, USA)

Clinical Cases and Perspectives on Diagnosis and Management of Pediatric Hypophosphatemic Rickets – Zulf Mughal (Dubai, UAE)

Panel Discussion and Audience Q & A – Agnès Linglart (Paris, France)

Sunday 23 June 2025

SUNDAY

Exhibition Hub Schedule

PROGRAMME to follow

 

SUNDAY 10:30-11:00

Patient Group Presentation

SUNDAY 13:10

Kyowa Kirin Meet The Expert Session

PROGRAMME to follow

 

SUNDAY 15:20

ISCBH Membership Presentation

SUNDAY 17:30-19:00

A Satellite Symposium organised by Alexion

A Leap Forward: Improving Diagnosis and Management of Pediatric Patients With Hypophosphatasia (HPP)

PROGRAMME

Welcome and introductions – Agnès Linglart (Paris, France)

Diagnostic criteria of HPP in children and adolescents – Oliver Semler (Cologne, Germany)

What we have learned: the clinical picture of HPP in children and adolescents from the global registry – Gabriel Ángel Martos-Moreno (Madrid, Spain)

Management of HPP in children and adolescents – Agnès Linglart (Paris, France)

Q&A – All

Wrap up/meeting close – All

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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