Lysosomal and Rare Disorders Research and Treatment Center - LDRTC

11/16/2025

Day 1 of GRIDS2025 is underway!

11/14/2025

There is still time to register!

Just Under 2 More Days till our 11th Annual GRIDSymposium 2025, hosted by LDRTC at Capital One Hall in Tysons, Virginia.

For more information and registration Click the link below:

GRIDS convenes global experts to address recent advances and challenges in lysosomal storage disorders and rare genetic diseases, fostering collaboration that drives innovation and improves patient outcomes.

11/04/2025

We’re excited to see you on Saturday!

EVENT DATE: Saturday, November 8th
TIME: 10:00 am to 4:00 pm

LOCATION:
DoubleTree by Hilton Richmond Midlothian
1021 Koger Center Boulevard, Richmond, VA 23235
Meeting Room Parlor FE

10/31/2025

As we close Gaucher Disease Awareness Month, our focus on advancing care, research, and education in Gaucher disease continues year-round.
We’re pleased to introduce the upcoming annual symposium GRIDS2025, themed “Innovations in Lysosomal Disorders”. The highlight following the symposium will be the GRIDS Clinic, where international Gaucher disease experts and radiologists will review select patient cases, exploring the broad spectrum of skeletal involvement in Gaucher disease through clinical and imaging perspectives. This interactive session offers an exceptional learning opportunity for: Trainees specializing in lysosomal and metabolic disorders, specialists seeking to deepen their understanding of the spectrum of bone pathology and radiologic assessment in Gaucher disease
Together, we continue to connect science, clinical care, and education to improve outcomes for those living with Gaucher disease.

10/31/2025

15 More Days till our 11th Annual GRIDSymposium 2025, hosted by LDRTC at Capital One Hall in Tysons, Virginia.

For more information and registration
Click the link below:

GRIDS convenes global experts to address recent advances and challenges in lysosomal storage disorders and rare genetic diseases, fostering collaboration that drives innovation and improves patient outcomes.

10/27/2025

We are delighted to invite you to the 11th Annual GRIDSymposium 2025, hosted by the Lysosomal & Rare Diseases Research Treatment Center (LDRTC).
This year’s event will take place on November 16th–17th at the Capital One Hall in Tysons, Virginia.

Keynote Speaker:
Behzad Najafian, MD
University of Washington, Seattle, WA, USA
Keynote Lecture: “AI in Medicine: Transforming the Landscape of Tissue-Based Diagnostics”

We look forward to your participation in this important gathering.

http://www.gridssymposium.com

10/20/2025

As we raise awareness this October, we reaffirm our commitment to advancing research, early detection, and innovative treatments that improve outcomes for individuals living with Gaucher disease and other lysosomal disorders.

10/20/2025

We are delighted to invite you to the 11th Annual GRIDSymposium 2025, hosted by the Lysosomal & Rare Diseases Research Treatment Center (LDRTC).
This year’s event will take place on November 16th–17th at the Capital One Hall in Tysons, Virginia.

Keynote Speaker:
Behzad Najafian, MD
University of Washington, Seattle, WA, USA
Keynote Lecture: “AI in Medicine: Transforming the Landscape of Tissue-Based Diagnostics”

We're looking forward to you taking part in this important gathering.

GRIDS convenes global experts to address recent advances and challenges in lysosomal storage disorders and rare genetic diseases, fostering collaboration that drives innovation and improves patient outcomes.

10/17/2025

🧬 New Publication from LDRTC: Enzyme Replacement Therapy in Infants and Very Young Children with Gaucher Disease using velaglucerase alfa: a single-center experience

We are sharing our latest publication, released in timely alignment with Gaucher Disease Awareness Month. This study, conducted at the Lysosomal & Rare Disorders Research & Treatment Center (LDRTC), presents real-world data on the use of ERT in infants and very young children — including several identified through newborn screening.

Our findings show that early ERT was well-tolerated and associated with rapid improvements in hematologic parameters, organ size, and biomarker profiles (notably Lyso-Gb1 levels) — emphasizing the vital importance of early diagnosis and intervention in Gaucher disease.

These results provide valuable insights into the early clinical course of Gaucher disease, underscoring how timely treatment can prevent irreversible complications and support normal growth and development.

Front. Pediatrics, 16 October 2025

Sec. Genetics of Common and Rare Diseases

Volume 13 - 2025 |

ObjectiveTo evaluate the effectiveness and safety of enzyme replacement therapy (ERT) with velaglucerase alfa, and offer insights into the clinical course of...

10/16/2025

We're onsite and ready to connect!

The LDRTC team is here to support the Fabry community, answering questions, providing resources, and sharing information about Fabry disease. Whether you're newly diagnosed, a caregiver, or just looking to learn more, we're happy to help. 💜

Stop by and say hello-we'd love to chat!

10/16/2025

Gaucher Disease Awareness Month

Early matters. When treatment begins—before Gaucher disease causes significant organ or bone changes—children can grow and thrive with healthy outcomes.

At LDRTC, we believe early diagnosis gives every child a stronger start and a brighter future.

10/07/2025

As we continue to observe Gaucher Disease Awareness Month, we turn our focus to patient advocacy. At LDRTC, we serve as dedicated partners in advancing equitable access to care. This includes advocating for access to current treatments through clinical trials, representing underrepresented voices in discussions about rare diseases, and fostering supportive networks for families.



We encourage you to share your experiences or significant advocacy milestones below. Your contributions are invaluable in strengthening our collective mission.



For more information: lysosomaltreatmentcenter.com or email info@Ldrtc.org