The Lysosomal Disorders Research and Treatment Center (LDRTC) supports individuals and families affected by lysosomal disorders, including Gaucher, Fabry, Pompe, Niemann-Pick, MPS, and related conditions. We combine patient advocacy, education, and research-focused care to help people navigate diagnosis, treatment, and daily life.

Our work is guided by five core priorities:

1) Patient and family education to support informed decisions about care and treatment.

2) Access to care and treatment through advocacy for equitable coverage, timely referrals, and clinical trial opportunities.

3) Community connections so patients and caregivers can share experiences and find support.

4) Research and innovation that improve diagnosis and accelerate new therapies for lysosomal disorders.

5) Patient voice and rights by elevating lived experience and promoting policies that improve quality of life and long-term outcomes.

Together, we aim to make a measurable difference for people living with lysosomal disorders—providing support today while driving progress toward more effective treatments and cures. If you are a patient, caregiver, clinician, or researcher, we welcome you to connect with us and learn more about our programs and services.