International Gaucher Alliance

23/08/2026

Join Us (call to action)

The IGA Volunteer Training Programme is opening its doors.
If you are part of the Gaucher community and want to build your advocacy skills, learn from patient leaders around the world, and grow into a stronger voice for your community, this is for you. No special background needed, just curiosity and a bit of time.

Ready to join, or want to know more? Write to Vesna at [email protected], and we will guide you from there.


Have a good day,

20/08/2026

🎥 NEW YOUTUBE VIDEO!

What does equity mean to you? 💬

At the Rare Diseases International meeting, we asked members of the rare disease community one important question: What is equity?

We heard different perspectives, experiences and ideas — all highlighting why equity matters when working towards a world where everyone affected by a rare disease has the opportunity to access the care, support and recognition they need.

🎥 Hear what our community had to say in our new YouTube video.

What does equity mean to you? 💬At the Rare Diseases International...

18/08/2026

The Importance of partnerships creates change for patients around the world 🌍

Sanofi’s Rare Humanitarian Program provides sustainable access to care for people with rare diseases based on medical need, regardless of where in the world they live or their ability to pay

13/08/2026

Yuhan has announced an important milestone in the development of its investigational Gaucher disease therapy, YH35995, after receiving Orphan Drug Designation from the European Medicines Agency (EMA). The designation follows orphan status granted by the U.S. FDA earlier this year and supports the continued global development of this oral therapy, which has shown potential to cross the blood–brain barrier—a key area of interest for neuronopathic (Type 3) Gaucher disease, where there remains a significant unmet need.

Yuhan Corp. said Tuesday that its investigational Gaucher disease therapy YH35995 has received orphan drug designation from the European Medicines Agency (EMA), adding to orphan status granted by t...

12/08/2026

Something new is on its way for our Gaucher volunteer community.
The IGA Volunteer Training Programme is a practical, self-paced path to grow your advocacy skills, connect with fellow volunteers across the world and step into greater leadership within your own community. Advocacy and leadership, patient group management, research and therapies, health policy, communications, and train-the-trainer — one theme at a time, built around the life you already have.
Watch this space. We will share how to get involved very soon.

07/08/2026

PRESS RELEASE

The International Gaucher Alliance and Gaucher Community Alliance are profoundly disappointed in Eli Lilly’s sudden and immediate termination of the PROCEED Type 1 Gaucher Gene Therapy Clinical Trial as part of “portfolio prioritization.”

The trial was terminated for financial reasons on the same day as the Eli Lilly CFO boasted of 48% revenue growth, multiple acquisitions, and raised full-year revenue guidance.

The cost of a rare disease clinical trial should not be a surprise to institutional pharmaceutical companies. When a company asks for the trust of a rare community, they must commit to seeing the trial through to then end, absent safety concerns.

To the patients and families that trusted Eli Lilly and enrolled in the trial, we honor your bravery and promise to ensure that this science will not be lost.

Eli Lilly, your corporate focus on profits over patients is heard loud and clear.

Tanya Collin-Histed
CEO, International Gaucher Alliance (IGA)

Aviva Rosenberg
CEO, Gaucher Community Alliance (GCA)
President, International Gaucher Alliance (IGA)

Cyndi Franks
President, Gaucher Community Alliance (GCA)

06/08/2026

Living well on treatment: new data on pain, fatigue and quality of life

A study published in late July has found that among adults already receiving treatment for Gaucher disease type 1, higher levels of pain and fatigue were still linked to poorer quality of life — even when standard clinical markers looked stable.
The findings support what many in our community have long said: being on therapy is not the same as feeling well. The researchers are calling for pain and fatigue to be tracked routinely alongside blood counts and organ measurements, using validated patient-reported outcome tools.
For our members, this is more than a research note. It is evidence you can bring to your own clinical team — a reason to ask that how you feel, not only your bloodwork, be part of every review.

Pain and fatigue are linked to poorer quality of life in adults with treated Gaucher type 1, a new study suggests.

31/07/2026

Our July Newsletter is here!
The first half of 2026 has been a busy and impactful one for the International Gaucher Alliance. This edition reflects on the difference we've made together—from global advocacy and education to research, collaboration and strengthening the voice of the Gaucher community around the world.

https://gaucheralliance.org/wp-content/uploads/2026/07/Webinar-newsletter-segments-4-1.pdf

23/07/2026

Mental health is an essential part of rare disease care, yet too many people are facing these challenges without the support they need. New findings from the latest Rare Barometer survey reveal the significant psychological impact of living with a rare disease and highlight the urgent need to integrate mental health into routine care.

Read more in our latest article: 🔗https://www.eurordis.org/from-evidence-to-action-closing-the-mental-health-gap-in-rare-disease-care/?fbclid=IwY2xjawTK1nRleHRuA2FlbQIxMABicmlkETBFRXZGTDRQQW9VVUZJaW9Uc3J0YwZhcHBfaWQQMjIyMDM5MTc4ODIwMDg5MgABHqKa8W3V6pozakx6nt54zdn1qrLMvtjkyojV5cSYbu18zciwAckB_QfK4KvC_aem_zSzIHNIqRQwG4oN-rLMocg

16/07/2026

Global experts unite to shape the future of GD3 clinical trials

Yesterday, the International Gaucher Alliance (IGA), in partnership with the International Working Group on Gaucher Disease (IWGGD), reached an exciting milestone in a collaborative international project to develop expert consensus on the design of future clinical trials for neuronopathic Gaucher disease (GD3).
Chaired by Professor Derralynn Hughes, the consensus meeting brought together leading GD3 clinicians and researchers from around the world, alongside experts in health technology assessment (HTA) and regulatory science. Together, they explored the key elements needed to ensure future GD3 clinical trials are scientifically robust, clinically meaningful, and capable of generating the evidence needed by regulators and HTA bodies as new therapies emerge.
This project is being led by a Steering Group comprising Professor Derralynn Hughes, Tanya Collin-Histed, Dr Sheela Upadhyaya, Dan O'Connor and Lucia Lavelle, working together to guide the Delphi process and build international consensus across the GD3 community.
The meeting represents one stage of the Delphi process. The discussions and recommendations will now be incorporated into the next round before a final consensus document is developed and submitted for publication. We hope this internationally agreed guidance will become an important reference for researchers, industry, regulators and clinicians, helping to accelerate the development of better-designed clinical trials and, ultimately, improved treatment options for people living with GD3 and their families worldwide.
The IGA would like to thank Professor Derralynn Hughes for expertly chairing the meeting, our Steering Group colleagues for their dedication, and every expert who generously contributed their time and knowledge. This project demonstrates the power of global collaboration, bringing together clinical, scientific, regulatory, HTA and patient leadership expertise with one shared goal—to improve the future for everyone affected by GD3.
We look forward to keeping our members updated as the project progresses.