The ASBMR 2025 Annual Meeting will take place from September 5–8, 2025, at the Seattle Convention Center, Seattle, Washington, USA. This meeting is the world’s leading international forum for bone, mineral and musculoskeletal research, bringing together more than 2,500 participants from over 50 countries, including clinicians, researchers, advocacy groups, patient organisations and healthcare professionals.

The program will feature around 100 scientific sessions and close to 1,000 poster presentations, covering a wide range of advances from fundamental science to clinical applications. A key highlight is the ASBMR/RBDA Pre-Meeting Symposium on Rare Bone Diseases: From the Genome to the Lived Experience, scheduled for September 4, 2025, which will focus on how cutting-edge science and the lived experience of people with rare bone conditions can converge to shape better care, research, and innovation.

ERBF contribution

The European Rare Bone Forum (ERBF) will be represented at this meeting through the participation of Catherine Nester, ERBF Board and Steering Group member. She will present a scientific poster entitled: “Accelerating Research and Development for Rare Bone Conditions in Europe: A Multistakeholder Call-to-Action.” ANDO Portugal, as a member of ERBF, played a key role in its realization, collaborating in the authorship of the abstract, submission, and preparation of the presentation and graphics of the poster.

This presentation is directly connected to the important work that ERBF has been leading since 2022, namely the development of a white paper addressing the challenges faced in research for rare bone conditions (RBCs). The white paper proposes concrete solutions to overcome barriers in the research and development (R&D) pathway and serves as a call to action to all RBC stakeholders — including patient organisations, healthcare professionals, researchers, industry, and policymakers.

The ERBF white paper was developed in collaboration with Costello Medical and highlights the need for collaborative, cross-sector approaches to accelerate research and bring meaningful impact to people living with rare bone conditions. Through this participation at the ASBMR 2025 Annual Meeting, ERBF strengthens its commitment to ensuring that the voices and needs of the rare bone community in Europe are represented in the global scientific arena.

It was officially launched during the Joint Congress of the European Society of Paediatric Endocrinology (ESPE) and the European Society of Endocrinology (ESE) on 11 May 2025.

Read the full white paper here: Research and Development of Treatments for Rare Bone Conditions in Europe

Stay tuned to learn more and to be updated on our work on Rare Bone Conditions.

Visit the European Rare Bone Forum for more news and information on Rare Bone Conditions here

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The European Rare Bone Forum (ERBF) is proud to announce the publication of its latest white paper: “Research and Development of Treatments for Rare Bone Conditions in Europe”, developed in collaboration with Costello Medical. This landmark document outlines key challenges and opportunities in advancing therapies for rare bone conditions (RBCs), and calls for stronger cross-sector collaboration, policy support, and investment in R&D.

The white paper was officially launched during the Joint Congress of the European Society of Paediatric Endocrinology (ESPE) and the European Society of Endocrinology (ESE) on 11 May 2025. Representing the ERBF and ANDO Portugal, Inês Alves presented the white paper and emphasized the need for a coordinated European strategy to improve diagnosis, treatment access, and innovation in this underrepresented field.

This white paper is the result of a multi-stakeholder collaboration, bringing together patients, clinicians, researchers, and industry representatives. It highlights the barriers to effective treatment development—such as data fragmentation, limited patient registries, and a lack of targeted funding—and proposes concrete recommendations to drive progress.

Read the full white paper here: Research and Development of Treatments for Rare Bone Conditions in Europe

Stay tuned to learn more and to be updated on our work on Rare Bone Conditions.

Visit the European Rare Bone Forum for more news and information on Rare Bone Conditions here

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On Jan 24, Ipsen announced the Canada approval of Sohonos (palovarotene capsules). Indicated to reduce new bone formation in adults and children (8 years and above for females and 10 years and above for males) with FOP. Sohonos is approved for both chronic use, and for flare-ups. It is the first approval for Sohonos worldwide.

FOP is a progressive and debilitating condition which has such a profound impact on patients, and their families. Until today, there was no approved medicine, and we are proud to bring this important new medicine to the FOP community

Dr. Howard Mayer, Executive Vice President and Head of Research and Development, Ipsen

About the MOVE clinical program

This approval is based on data from the MOVE trial. The first global multi-center Phase III trial in FOP. MOVE is an open-label, single-arm trial, evaluating the efficacy and safety of palovarotene in decreasing new ossification volume in patients with FOP.

About Sohonos

Sohonos is an oral, selective RARγ agonist developed as a treatment for people living with this debilitating ultra-rare genetic disorder. The treatment was acquired by Ipsen through the acquisition of Clementia Pharmaceuticals.

About Ipsen

Ipsen is a global biopharmaceutical company focused on transformative medicines in Oncology, Rare Disease and Neuroscience; For more information, visit ipsen.com.

Source:

Business Wire

Visit the European Rare Bone Forum (ERBF) for more news and information on Rare Bone Conditions here

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The European Rare Bone Forum Call To Action Project kicks off on January 2022. Visit rarebone.org for more information on this project which aims to improve access to treatments for Rare Bone Diseases and to influence policies at the national and European level.

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A recently published study entitled Patients’ priorities and expectations on an EU registry for rare bone and mineral conditions(1), with the participation of Inês Alves from ANDO Portugal (Patient Group Representative of the European Rare Bone Forum), highlights the importance of understanding the natural history of rare bone and mineral conditions to improve clinical practice and the development of new diagnostics and therapies(2). Additionally, the article also states that recruitment and long-term participation in registries are key challenges for researchers.

In brief, the European Patient Advocacy Group (or ePAGs) of the European Reference Network for Rare Bone Diseases — ERN BON(3) works together with other patient organisations, clinicians, experts and researchers for the development of the European Registry for Rare Bone and Mineral Diseases, which provides networks for learning and research collaboration between industry, scientific researchers, regulators, clinicians, patient organisations and families.

In the end, these registries provide an understanding of the natural history of diseases (especially rare ones) in terms of the type and severity of complications, progression, impact on quality of life, risk-benefit ratio and other clinical parameters, and are essential for improving people’s treatment pathways and the development of new diagnostics and therapies.

How was the study conducted?

In order to understand user needs, the European Reference Network for Rare Bone Diseases – ERN BOND(3) and the European Patient Advocacy Groups developed and implemented a multinational survey on the content and functionality of the database of choice for participants, through an iterative consensus process. The survey was disseminated by national and international patient and health professional groups, and results were analysed using descriptive statistics and multivariate regression.

What were the results?

There were 493 responses from 378 adults, 15 children and 100 parents, guardians or carers (PTCs) with 22 different rare bone and mineral conditions. The survey was conducted online in several languages, first released in English and then translated and proofread by native speakers of Czech, French, Dutch, Estonian, German, Italian, Portuguese and Swedish.

Results to note:

• Content on anxiety and socialisation scored the lowest
• Additional content was recommended by 205 respondents
• Respondents preferred data entry by their healthcare provider (HCP)
• Less than 50% of adults received follow-up care from their HCP at least annually
• 29% of adults responded that they were followed as needed

Table 1 — Rare bone and Mineral condition type of respondent

Table 2 — Interest in the features of the proposed database

Findings

• This survey of individuals, their families, guardians and carers prioritised key components to create a research database for rare bone and mineral conditions in the European Union.
• The survey highlights issues related to the collection of psychosocial impacts as well as an analysis of levels of trust in the health care provider.
• The survey showed that only visits to specialised centres for data collection, although preferred by patients, will be insufficient for a substantial number of people, limiting generalisation.
• Combined healthcare provider and patient platforms will be needed to collect representative and complete natural history data for this patient group.

Know more about the ERBF Members, Mission and visit our website: rarebone.org

References

1. Javaid MK, et al. Patients’ priorities and expectations on an EU registry for rare bone and mineral conditions. Orphanet J Rare Dis. 2021;16(1):463. Published 2021 Nov 3. doi:10.1186/s13023-021-02069-9
2. Administration USFaD. Rare Diseases: Natural History Studies for Drug Development—Draft Guidance. In: Office of Medical Products and Tobacco OoSMP, Office of Orphan Products Development, Office of Medical Products and Tobacco, Center for Drug Evaluation and Research, Office of Medical Products and Tobacco, Center for Biologics Evaluation and Research, editor. 2019.
3. Heon-Klin V. European Reference networks for rare diseases: what is the conceptual framework? Orphanet J Rare Dis. 2017;12(1):137. doi: 10.1186/s13023-017-0676-3.

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