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Patient Group Members

About

The ISCBH works closely with our Patient Group Members to strengthen collaboration with organisations supporting children’s bone health.

We have a new Patient Group Committee which will help shape patient-centred initiatives at the ISCBH. These partnerships provide a platform to amplify the patient voice which is so important in our field. 

For Patient Groups

If you are a person with lived experience and are interested in becoming a Patient Group Member of the ISCBH, we would love to hear from you. Please get in touch by email to info@theiscbh.org and we will send you information.

Patient Group Consultancy Service

The Patient Group Consultancy service can help our members incorporate the patient voice on clinical, quality improvement, or research related matters by offering advice and guidance on how best to achieve their goals.  Requests for help are reviewed by the Chair of the Patient Group Committee, after which the Patient Group Committee can provide specific advice to the member.  You must be a member to use this service. To find out more, please contact us at info@theiscbh.org.

ISCBH Patient Group Members

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The Osteogenesis Imperfecta Foundation, Inc. (OI Foundation) is the only voluntary national health organization dedicated to helping people cope with the problems associated with osteogenesis imperfecta. Our mission is to improve the quality of life for people affected by OI through research, education, awareness, and mutual support. The OI Foundation began in 1970 by a small group of parents who banded together to stimulate public and professional interest, support families and encourage research. Today, the OIF provides programs and services for OI community members and medical professionals including the online OIF Information Center (medically verified by OIF’s Medical Advisory Council), National and Regional conferences, Clinic and provider directories, Support Networks, Impact Grants, and the OI Registry.

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Osteogenesis Imperfecta Federation Europe (OIFE) is an umbrella association for organizations dealing with the rare genetic bone condition osteogenesis imperfecta (OI). OI is caused by a defect in the production of collagen.

OIFE was first established in 1993 in the Netherlands. From 2022 we have been registered as a non profit in Belgium. It’s mission is to connect and empower organizations, professionals and individuals to improve lives of people with OI. Our vision is children and adults with OI living active and independent lives – with access to competent healthcare and necessary social support.

OIFE’s ordinary member organizations are European and our main activities are based in Europe. But we collaborate with OI-organizations around the world with the intention to exchange ideas, information and best practices. OIFE include 41 member organizations:

  • 20 European national organizations
  • 16 partner organizations – national OI-organizations outside Europe
  • 5 supporting organizations

OIFE is a member of EURORDIS – the umbrella for rare disease organizations in Europe, the European Rare Bone Forum and an affiliated member of the European Society of Endocrinology (ESE).

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Canadian XLH Network

BKMF e.V. offers support for short-statured people and their families with the focus on “helping people themselves”. The non-profit organization has 3.500 members with 90 different conditions. Our goal is to inform with evidence based information material ensured by our academic advisory board. We participate and encourage research projects. BKMF e.V. offers counseling by voluntary and full-time advisors. There is a broad range of different seminars ranging from a seminar for new parents, youth seminars, sport events, and the Kleinwuchsforum, our annual convention. It has 1.000 attendees and is the largest event of its kind in Europe. Overall, there are roughly 10 seminars per year on a federal level.

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The Canadian XLH Network is a national, patient-led organization dedicated to improving the lives of individuals and families affected by X-linked hypophosphatemia (XLH), a rare, lifelong metabolic bone disorder. At its core, the Network is built on the values of collaboration, education, and advocacy. By uniting patients, families, caregivers, and health care professionals, the Network works tirelessly to raise awareness of XLH and its challenges, ensuring that those living with the condition are not navigating it alone.

The mission of the Canadian XLH Network is to advocate for equal and timely access to the best available treatments and care across the country. The Network promotes education by providing resources and opportunities to learn—not only for affected families but also for the broader medical community and the public—fostering greater understanding of this rare disease.

Through partnerships with like-minded organizations, both nationally and internationally, the Canadian XLH Network amplifies the voices of the rare disease community. Just as importantly, it offers support and connection to patients and families, helping them cope with the physical, emotional, and social impacts of XLH. Together, the Canadian XLH Network strives to build a stronger, more informed, and more compassionate future for all Canadians living with XLH.

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The International FOP Association (IFOPA) is a global community dedicated to helping individuals and families living with Fibrodysplasia Ossificans Progressiva (FOP). FOP is an ultra-rare genetic condition where muscle and connective tissue slowly turn into bone, making movement progressively more difficult. Since 1988, when FOP patient Jeannie Peeper founded the IFOPA, the organization has been a source of hope, information, and connection for people around the world.

At the heart of the IFOPA’s mission is the belief that no one should face FOP alone.  The IFOPA provides education to help families understand and manage the condition, while also sharing valuable resources with doctors and caregivers. Through support programs such as peer mentoring, online groups, family guidebooks, and financial assistance, the IFOPA helps ease everyday challenges and builds stronger connections among those affected. The IFOPA also invests in promising research and works hand-in-hand with scientists to accelerate progress toward better treatments and, one day, a cure.

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FOP Friends is a UK-based charity dedicated to providing support, information, and funding for the community affected by Fibrodysplasia Ossificans Progressiva (FOP).  

FOP is an ultra-rare and devastating genetic disorder where muscles, tendons, and ligaments gradually turn into bone, leading to progressive, permanent immobility.
The charity works tirelessly to improve the lives of individuals living with FOP and their families. Including:

  • Raising awareness of this debilitating condition among the public and medical professionals.
  • Funding research worldwide to find effective treatments and, ultimately, a cure.
  • Providing practical and emotional support to those affected, helping them navigate the complexities of living with FOP.

FOP Friends offers a vital lifeline to a small but strong community, ensuring that FOP is not forgotten and that the quest for a cure continues.

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The XLH Network is a national nonprofit organization dedicated to supporting individuals and families affected by X-linked Hypophosphatemia (XLH), a rare genetic disorder characterized by impaired phosphate metabolism and skeletal abnormalities. Our mission is to advance patient care, promote disease awareness, and facilitate collaboration between patients, caregivers, and healthcare professionals.

We provide comprehensive educational resources, including webinars, workshops, and clinical materials, designed to support both patients and medical providers in understanding the complexities of XLH. The Network also offers specialized programs for children and adolescents, fostering engagement, resilience, and peer support through initiatives such as the XLH Pup Squad.

In addition to patient and caregiver support, the XLH Network actively partners with clinicians, researchers, and pharmaceutical stakeholders to advance research, improve clinical outcomes, and inform best practices. We facilitate opportunities for patient participation in clinical studies and advocate for timely access to emerging therapies and multidisciplinary care.

By combining education, advocacy, and professional collaboration, the XLH Network serves as a central resource for the XLH community, empowering patients while promoting evidence-based care, advancing research, and supporting the broader healthcare ecosystem in delivering optimal management for this rare condition.

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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.

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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.

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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.

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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.

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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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