International Gaucher Alliance

International Gaucher Alliance The IGA is an international umbrella group representing the interest of Gaucher Patients and non for profit Gaucher Patient Groups throughout the world.

The IGA is an international umbrella group representing the interests of Gaucher patients and those of non-for-profit Gaucher patient groups as well as rare disease groups throughout the world. Why do we need an international umbrella group? The working together of Gaucher patient groups has already lead to consistency in treatment of certain manifestations of Gaucher Disease throughout Europe. In

spired by this and by their successes in achieving humanitarian aid for the most severe patients in Eastern Europe member countries of the IGA have grasped the challenge of collaboration for the further advantage of Gaucher Patients. Although the most common Lysosomal Storage Disorder Gaucher Disease is very rare with an incidence of about 1 in 100,000 live births. Patient Groups have worked closely together to share their experiences and to facilitate access to treatment. Scientists and Clinicians throughout the world increasingly collaborate both in research and in the development of strategies for the management of the disease. Pharmaceutical companies are Global and clinical trials are frequently multi-centred with treatments being approved in different countries worldwide, while in others patients are not able to benefit from them. Patient Groups throughout the world cannot work in isolation and their alliance by joining the IGA provides a clear global voice of representation and opportunity for advancement.

Attending the European Conference on Rare Diseases (ECRD) in Prague has been an incredible opportunity for the Internati...
18/06/2026

Attending the European Conference on Rare Diseases (ECRD) in Prague has been an incredible opportunity for the International Gaucher Alliance (IGA) to strengthen our network, showcase our work, and explore new opportunities for collaboration.

One of the highlights was meeting with our colleagues from the Czech Gaucher Organisation, as well as Vit Hubeny and Katerina. These face-to-face conversations are invaluable for sharing knowledge, exchanging ideas, planning future initiatives, and strengthening partnerships across the rare disease community.

Working together is key to driving progress and ensuring that the voices of people living with Gaucher disease continue to be heard. We look forward to building on these connections and collaborating on future projects.

Representing the Global L*D Collaborative at the International MPS Network MeetingLast week, I attended the Internationa...
15/06/2026

Representing the Global L*D Collaborative at the International MPS Network Meeting

Last week, I attended the International MPS Network Meeting in Florence, Italy, representing the Global L*D Collaborative. The event brought together MPS patient organisations, researchers, families, and young people from around the world to share knowledge, experiences, and future priorities. I participated in a panel discussion exploring the future of the MPS community and how rare disease organisations can work together to improve diagnosis, access to treatment, clinical care, research, and long-term outcomes for patients and families.

During the discussion, I shared perspectives from the Gaucher community on the importance of meaningful patient involvement in research and registry governance. We discussed the value of patient-centred registries, the role of patient-reported outcomes, and the challenges of transitioning patients from paediatric to adult healthcare services. A particular highlight was meeting families and young adults living with MPS. Their experiences and achievements were a powerful reminder of the resilience of the rare disease community and the importance of ensuring patient voices remain at the centre of decision-making.

The meeting highlighted opportunities for greater collaboration across rare disease communities, including improving diagnosis, reducing inequalities in care, strengthening data collection, and sharing knowledge and expertise. The IGA thanks the International MPS Network for the invitation and looks forward to continuing to work together to improve outcomes for patients and families worldwide.

*DCollaborative

We are very happy to share that the International Gaucher Alliance (IGA) is participating in ECRD 2026 — the European Co...
04/06/2026

We are very happy to share that the International Gaucher Alliance (IGA) is participating in ECRD 2026 — the European Conference on Rare Diseases and Orphan Products, taking place on 3–4 June 2026 in Prague.

ECRD is a vital platform for shaping policies to address the unmet needs of people living with rare diseases. For the global Gaucher community, this is an important opportunity to showcase how we are actively bridging the gap between scientific research and daily patient care.

Our collaborative poster with the International Working Group on Gaucher Disease (IWGGD), titled "Bridging Worlds: How Collaboration Between Patient Organisations and Medical Professionals Is Transforming Gaucher Care Globally," demonstrates our joint commitment to building capacity and sustainability. Through initiatives like the Global Gaucher Connect Programme, the Africa Education Series, and regional partnerships like CEPCAL, we are working to ensure that accurate information and sustainable expertise are accessible worldwide.

At the IGA, we believe that patients and families must be partners in shaping the future. We look forward to connecting, learning, and representing the Gaucher community to advocate for timely, compassionate diagnosis, care, and treatment across the globe.

Together, we can build an inclusive, sustainable rare disease network where no matter where a patient lives, their needs are met.

EURORDIS-Rare Diseases Europe


Our May 2026 IGA Newsletter is now available to view on our website. This special IWGGD edition features scientific high...
28/05/2026

Our May 2026 IGA Newsletter is now available to view on our website. This special IWGGD edition features scientific highlights, community reflections, working group updates, interviews, and global perspectives from the 3rd IWGGD Symposium in Trieste. We hope you enjoy reading and thank everyone who contributed.

https://gaucheralliance.org/wp-content/uploads/2026/05/Webinar-newsletter-segments-2-1.pdf

Sanofi’s investigational therapy venglustat has been granted FDA Priority Review for the treatment of Type 3 Gaucher dis...
28/05/2026

Sanofi’s investigational therapy venglustat has been granted FDA Priority Review for the treatment of Type 3 Gaucher disease (GD3).

If approved, venglustat could become the first treatment in the US specifically targeting the neurological manifestations associated with GD3. The application is supported by positive Phase 3 LEAP2MONO study results, with the therapy showing potential to address neurological symptoms by crossing the blood-brain barrier.

This marks an important milestone for the neuronopathic Gaucher disease community and highlights continued progress in research, innovation, and future treatment possibilities for people living with GD3 worldwide. Read more at https://ml-eu.globenewswire.com/Resource/Download/b55d4f71-7a77-4c06-a14e-0285adceca63

21/05/2026

Andre Balzekiene spoke with Phoebe Wamalwa about her work, expertise, and insights from the Gaucher community at this year’s IWGGD meeting 💙

We believe that patient participation and collaboration are at the heart of improving outcomes for people living with Ga...
14/05/2026

We believe that patient participation and collaboration are at the heart of improving outcomes for people living with Gaucher disease. Our work alongside the International Working Group on Gaucher Disease is built on a shared commitment to making sure the patient voice is heard and included across research, education, clinical care, and wider discussions around policy and access to treatment.
By working together with clinicians, researchers, patient advocates, and industry partners, we can better understand the real challenges faced by individuals and families affected by Gaucher disease. Patient insight plays a vital role in shaping priorities, identifying unmet needs, and helping ensure that initiatives are grounded in lived experience. Through continued collaboration, we hope to build a more connected and supportive global community, one that encourages innovation while keeping patients at the centre of every conversation.

We are happy to share that poster was awarded at the 3rd IWGGD symposium in Trieste. The campaign marked a month of aw...
06/05/2026

We are happy to share that poster was awarded at the 3rd IWGGD symposium in Trieste. The campaign marked a month of awareness and reached 2 million people globally. A sincere gratitude to the Gaucher community who contributed and shared their stories.

The IGA family at the IWGGD symposium in Trieste.
05/05/2026

The IGA family at the IWGGD symposium in Trieste.

The first session of the IWGGD Symposium — patient session, highlights the importance of lived experiences in shaping be...
04/05/2026

The first session of the IWGGD Symposium — patient session, highlights the importance of lived experiences in shaping better care.
This year, in collaboration with the (IGA), the spotlight was on Africa and global reach—bringing forward diverse perspectives and shared challenges from across regions.
Presentations featured: Chris Hendriksz (United Kingdom), Pheobe Wamalwa (Kenya), Kandi-Catherine Muze (Tanzania),Roselyn Karungari Kanja (Kenya), Vesna Aleksovska (North Macedonia), Carolina Toneloto (Brazil). Session was moderated by IGA’s CEO Tanya Collin-Histed.

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