Lysosomal and Rare Disorders Research and Treatment Center - LDRTC

04/02/2026

April is Fabry Disease Awareness Month.
Fabry disease is a rare genetic condition— and is frequently missed for years. At LDRTC, we have been following patients with Fabry disease, some over a decade. What makes the difference: early diagnosis, uninterrupted care, and access to the latest therapies. The treatment landscape is constantly evolving.

03/27/2026

Register for Upcoming CME Activity: Lysosomal Disorders and the Heart!
Here: https://us06web.zoom.us/webinar/register/WN_GxfbPqzsSbW-7ZCr0hUOPg #/registration

02/28/2026

“Rare Disease Day emphasizes collaboration with patients and their communities to advance the development of treatments for rare diseases.”
The message from CMO of LDRTC.

02/28/2026

Happy Rare Disease Day from all of us at LDRTC!
To our patients and families near and far: you are the reason we do what we do. We see you, we stand with you, and be always with you in this journey.

02/27/2026

🌍 Over 300 million people worldwide live with a rare disease.
By raising awareness, we amplify their voices and support their journeys.
💙 At LDRTC, we stand with individuals and families around the world.

02/20/2026

Register for our upcoming "Webinar: Why S*x Matters in Rare Diseases" for free!
Wednesday, February 25th 7:30 PM ET / 4:30 PM PT
More information here:

02/13/2026

Register for our upcoming "Webinar: Why S*x Matters in Rare Diseases" for free!
Wednesday, February 25th 7:30 PM ET / 4:30 PM PT More information here: https://gauchercommunity.app.neoncrm.com/nx/portal/neonevents/events?path=%2Fportal%2Fevents%2F36571

02/05/2026

Our Commitment to the Lysosomal Diseases Community
We are honored to support the Lysosomal Diseases community. Please visit our team at the exhibition hall to connect and learn more about our ongoing efforts and initiatives.

Featured Poster Session
Additionally, we invite you to attend Dr. Goker-Alpan’s poster session today, Thursday, February 5, 2026, at 3:30 PM. Dr. Goker-Alpan will present on “Prospective Dose Escalation Study with Ambroxol in Patients with Sanfilippo Syndrome.”

02/04/2026

Wednesday, February 4, 2026 | 6:45–7:45 AM
Beyond Storage: Secondary Glycosphingolipid Pathology in Lysosomal Disorders
CE Satellite Symposium

More Information: https://worldsymposia.org/worldsymposium-program-events-lysosomal-disease/satellite-symposia-worldsymposium/

02/04/2026

Join us for our Satellite Symposium happening now in Grand Ballroom CD. Beyond Storage: Secondary Glycosphingolipid pathology in Lysosomal Disorders.

02/03/2026

Wednesday, February 4, 2026 | 6:45–7:45 AM
Beyond Storage: Secondary Glycosphingolipid Pathology in Lysosomal Disorders
CE Satellite Symposium

Accredited Provider: AffinityCE
Jointly Provided By: AffinityCE and Lysosomal & Rare Disorders Research & Treatment Center, Inc. (LDRTC)

More Information: https://worldsymposia.org/worldsymposium-program-events-lysosomal-disease/satellite-symposia-worldsymposium/

02/01/2026

The LDRTC Team will be at the 22nd Annual WORLDSymposium Event. Taken place February 2nd to the 6th.

More information can be found at :

About the 22nd Annual WORLDSymposium™ WORLDSymposium will be conducting the Annual Research Meeting as a LIVE event. The meeting will be held at the , in , , USA.  On Demand recordings will be available for remote attendees and will include many of the core elements of the in-person Annual Resear...