Lysosomal and Rare Disorders Research and Treatment Center - LDRTC

10/15/2026

Just 2 days left! GRIDS 2026 brings together researchers, clinicians, and regulatory experts to discuss the path from research to approved therapies for rare diseases. Register now: https://gridssymposium.com/

Abstract submissions are open for platform and poster presentations in Melbourne, Florida, October 18–19, 2026. Share your research and help advance care for lysosomal disorders. Submit now.

10/14/2026

3 days to go! At GRIDS 2026, experts will explore how biomarkers are helping track disease progression and guide safer, more effective treatments for rare diseases. Join us virtually: https://gridssymposium.com/

Abstract submissions are open for platform and poster presentations in Melbourne, Florida, October 18–19, 2026. Share your research and help advance care for lysosomal disorders. Submit now.

10/13/2026

Continuing our recognition of Gaucher Disease Month,
Dr. Goker-Alpan is presenting the management of Neuronopathic Gaucher disease at the GCA Patient & Family Conference in Phoenix, Arizona. She looks forward to connecting with patients and families and sharing meaningful insights to support them throughout their care journey.

10/12/2026

Happening now: Dr. Ozlem Goker-Alpan presents “Artificial Intelligence and Gaucher Disease” at the Gaucher Community Alliance Conference, on how AI may support earlier diagnosis, clearer measurements, and individualized research.

10/12/2026

Only 5 days to go! There’s still time to join us virtually. Join GRIDS 2026 to explore patient-centered trial design, biomarkers, regulatory insights, and emerging therapies shaping safer, longer-lasting treatments for rare diseases. Register today and secure your spot! https://gridssymposium.com/

10/05/2026

Research at LDRTC offers encouraging evidence that infants and very young children who began enzyme replacement therapy before developing a significant disease burden maintained healthy growth, organ function, and stable biomarkers over time. These
promising findings highlight the life-changing potential of newborn screening, early diagnosis, and timely treatment. We’re also proud to collaborate with GCA in meaningful ways to support and strengthen the Gaucher community. We’re delighted to share that Dr. Ozlem Goker-Alpan will be presenting at the Gaucher Community Patient & Family Conference in Phoenix, Arizona. We hope you’ll join her for these engaging and informative sessions!
 Monday 10/12/2026 – “Artificial Intelligence and Rare Disease Research” - 1:50 PM
 Tuesday, 10/13/2026 – “Neuronopathic Gaucher Disease Discussion” – 1:00 PM

10/02/2026

Happy Gaucher Disease Awareness Month! This October we celebrate the strength and resilience of the Gaucher community: the patients, families, clinicians, researchers and advocates who make progress possible. It is also a moment to share, with pride, a look back on a busy year at LDRTC.

We continued our patient and family education meetings, including a workshop for parents of young children (April 2025) and another for young adults (April 2026). Our team published new research on early treatment in infants, early brain biomarkers in neuronopathic Gaucher disease, and bone health in women with Gaucher disease, and presented our work at IWGGD in Trieste and SSIEM 2026 in Helsinki.

We also continued our collaboration with the Gaucher Community Alliance (GCA) to advocate for newborn screening, so that every baby in every state can be diagnosed early, and we continued to expand access to new therapies through clinical trials, including gene therapy.

Thank you to our patients, families, and partners.

09/25/2026

Dr. Ozlem Goker-Alpan shares insights on biomarkers in neuropathic Gaucher disease at RHLF in Paris.

09/21/2026

The countdown is on! Just under 30 days until GRIDS Symposium!

Registration is still open and hotel rooms are available.

And yes, ask us about the complimentary shuttle from the airport to the venue.

You won't want to miss this one.
Register now:

Program: GRIDS2026 will provide a multidisciplinary, case-based program on trial design, endpoint selection, biomarker interpretation, safety monitoring, and post-trial implementation. with focused content on Gaucher disease, Fabry disease, Pompe disease, MPS and ASMD.

09/19/2026

Together, the LDRTC team is proud to advance Fabry disease research and expand meaningful outreach—helping build a stronger, more connected Fabry community.