Gaucher Community Alliance

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Don't forget to register for our upcoming webinar!🗓 Thursday, June 18th⏰ 7:00 PM ET / 4:00 PM PTPresented by:Pramod Mist...
06/08/2026

Don't forget to register for our upcoming webinar!

đź—“ Thursday, June 18th
⏰ 7:00 PM ET / 4:00 PM PT

Presented by:
Pramod Mistry, MBBS, PhD, MA, MD

During this session, Dr. Pramod Mistry will provide an overview of the design and key findings from a Phase 3 clinical study, which evaluated an investigational oral treatment called venglustat in adults and teenagers living with type 3 Gaucher disease. This webinar is intended to help patients and families better understand ongoing research efforts in the type 3 neuronopathic community.

Register here: https://gauchercommunity.app.neoncrm.com/nx/portal/neonevents/events?path=%2Fportal%2Fevents%2F45338

The FDA has granted priority review for venglustat for the treatment of type 3 Gaucher disease (GD3). If approved, veng...
05/29/2026

The FDA has granted priority review for venglustat for the treatment of type 3 Gaucher disease (GD3). If approved, venglustat would become the first treatment available in the US to address the progressive neurological manifestations associated with GD3. The target action date for the FDA decision is November 25, 2026.

For more details, see the press release from Sanofi: https://www.news.sanofi.us/2026-05-28-Sanofis-venglustat-accepted-for-priority-review-in-the-US-to-treat-type-3-Gaucher-disease

Registration is open for the 2026 Gaucher Community Patient & Family Conference!This national conference is designed to ...
05/28/2026

Registration is open for the 2026 Gaucher Community Patient & Family Conference!

This national conference is designed to connect, educate, and empower Gaucher patients and their families by providing information and tools to best manage Gaucher disease and live fuller lives by connecting with and learning from patients, industry partners, medical experts, and community peers.

Learn more and register on our website!

gauchercommunity.org

Cyndi Frank, President of the Gaucher Community Alliance, attended the GBA1 conference in Phoenix, AZ this past week. Th...
05/27/2026

Cyndi Frank, President of the Gaucher Community Alliance, attended the GBA1 conference in Phoenix, AZ this past week. The GBA1 conference is designed for patents, health professionals and stakeholders from both academia and industry who are involved in or specialize in GBA1 research and the connection between Gaucher disease and Parkinson’s disease. Topics covered all aspects of GBA1, ranging from basic science to clinical trials, and provided a unique platform to collaboratively advance the understanding and treatment of GBA1-associated neuro-degenerative diseases.

Pictured is: Deborah and Greg Macres of the Children’s Gaucher Research Fund and Cyndi Frank, President of the Gaucher Community Alliance

Learn more here: https://www.gba1conference.com/about-us/

Yesterday, the Gaucher Community Alliance launched a brand new and improved website! In addition to new features such as...
05/22/2026

Yesterday, the Gaucher Community Alliance launched a brand new and improved website!

In addition to new features such as an Events Calendar and a Photo Gallery, we published the official registration page for the 2026 Gaucher Community Patient & Family Conference! While you're exploring the new website, be sure to register for the upcoming conference as well -- we are looking forward to seeing you in Phoenix!

https://www.gauchercommunity.org/

Gaucher Women on the Hill -
05/19/2026

Gaucher Women on the Hill -

It's not too late to join this year's Washington DC Advocacy Trip: Women on the Hill! 🗓 June 24–June 26📍 Washington, DCT...
05/15/2026

It's not too late to join this year's Washington DC Advocacy Trip: Women on the Hill!

🗓 June 24–June 26
đź“Ť Washington, DC

This trip is designed to encourage advocacy within the Gaucher community, support meaningful steps toward policy changes that impact the Gaucher and rare disease communities, and create a women’s cohort to discuss issues unique to our health.

Register here: https://forms.gle/ECymWuMkk4uwuJKP8

Webinar: Updates from the Venglustat Clinical Trial in Type 3 GD Patients🗓 Thursday, June 18th⏰ 7:00 PM ET / 4:00 PM PTP...
05/12/2026

Webinar: Updates from the Venglustat Clinical Trial in Type 3 GD Patients

đź—“ Thursday, June 18th
⏰ 7:00 PM ET / 4:00 PM PT

Presented by:
Pramod Mistry, MBBS, PhD, MA, MD

Professor of Medicine (Digestive Diseases) and of Pediatrics (Gastroenterology)
Professor of Cellular & Molecular Physiology
Director of Yale Lysosomal Disease Center and Gaucher Disease Treatment Center

Register here: https://gauchercommunity.app.neoncrm.com/nx/portal/neonevents/events?path=%2Fportal%2Fevents%2F45338

This educational program is sponsored by Sanofi. Venglustat is an investigational treatment and has not been approved by the FDA or any other regulatory authority. The information presented is not intended as medical advice. Please consult your physician regarding your individual medical condition and treatment options.

*Closed caption translation services available.

Aviva had a lovely week attending IWGGD Symposium in Trieste, Italy. (International Working Group on Gaucher Disease). E...
05/05/2026

Aviva had a lovely week attending IWGGD Symposium in Trieste, Italy. (International Working Group on Gaucher Disease). Experts from around the world presented on topics from Gaucher diagnosis in Africa to skin manifestations to Gcase modifiers. Aviva presented a poster on newborn screening challenges in the United States. It was an inspiring week meeting incredible patients from around the world.

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Pittsburgh, PA

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+14122199188

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