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FOUNDING CORPORATE PARTNER

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.  Visit our website to learn more.

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Corporate Partners

Kyowa Kirin Logo
FOUNDING CORPORATE PARTNER

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. You can learn more about the business of Kyowa Kirin on our website.

*Harmony and loop among people.

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FOUNDING CORPORATE PARTNER

Mereo BioPharma is a biopharmaceutical company focused on the development of innovative therapeutics for rare diseases. We are driven by a true understanding of the critical nature of our mission, we harness the power of science and meaningful relationships to create new possibilities for patients who need them. The Mereo team is a crew on a mission. We understand the gravity of our work for people with few therapeutic options, and that as such, there is no room for passengers. Every single team member is here because of their passion for helping people with health conditions, and their unique skills, experience and perspectives. We have two rare disease product candidates, one for the treatment of osteogenesis imperfecta (OI) and one primarily for the treatment of severe alpha-1-antitrypsin deficiency-associated lung disease (AATD-LD). In addition to the rare disease programs, Mereo has two oncology product candidates in clinical development.

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Registered Office: Kaiser Josef Straße 9, 6020 Innsbruck, Austria

© 2023 International Society for Children’s Bone Health

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.

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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.

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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.

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