Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)

About

LDRTC is a non-profit organization founded in 2013 by Dr. Ozlem Goker-Alpan with a vision to provide high-quality care for individuals with lysosomal storage diseases and other rare genetic disorders. Dr. Ozlem Goker-Alpan is a world-renowned authority in the field of lysosomal and other rare genetic disorders and is well known for her dedication to the health and well-being of her patients and families.

Our Goal:
LDRTC utilizes a variety of clinical treatment and research protocols, conducts training programs to increase awareness, and offers compassionate care and a platform for the patients and families affected by these debilitating disorders.

Our Commitment:
Research to unravel underlying disease-causing mechanisms, discover new biomarkers, and explore new therapeutic pathways and treatment options.
Education for patients, families, healthcare providers, and researchers in the field. Treatment and clinical care for persons suffering from Lysosomal and other Rare diseases.

Our Approach:
LDRTC aims to bridge the gap between basic and clinical research to facilitate the diagnosis, treatment, and management of rare disorders. LDRTC presents a unique translational research model focusing on a multidisciplinary approach to study the disease pathology while applying cellular, molecular and immunologic concepts to pathways amenable to therapeutic modulation.

Our goal is to implement personalized medicine wherein we recognize and acknowledge unique medical histories presented by individuals with rare disorders and ultimately offer medical care. We believe novel treatment options are worth exploring even if they benefit as few as a single patient. We design and offer true "Bench to Bedside" clinical protocols where there is a need to study a disease aspect or a mechanism for which there is an already existing therapy (drug re-purposing) or a pipeline product.

Keywords

genetic diseases, autophagy, mucopolysaccharidosis, pompe disease, lysosomal storage disorders, lysosomal storage diseases, rare diseases, bone disease, gaucher disease, enzyme replacement therapy, substrate reduction therapy, niemann pick disease, fabry disease, inherited metabolic disorders

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