Lysosomal Disease Network
At a Glance
- Status: Active Consortium
- Year Launched: 2004
- Initiating Organization: NIH Rare Disease Clinical Research Network
- Initiator Type: Government
- Rare disease
- Disease focus:
Alpha-Mannosidosis Types I / Ii, Aspartylglucosaminuria, Batten disease, Batten disease late infantile, Beta-mannosidosis, Cystinosis, Danon disease, Fabry disease, Farber disease, Fucosidosis, Galactosialidosis types I/II, Gaucher disease, Hunter syndrome, GM1-Gangliosidosis types I/II/III, GM2-Gangliosidosis, Hurler syndrome, I-cell disease, Krabbe disease, Maroteaux-Lamy syndrome, Metachromatic leukodystrophy, Morquio syndrome, Mucolipidosis type IV, Mucopolysa, Multiple sulfatase deficiency, Niemann-Pick disease, Northern Epilepsy, Pompe disease, psuedo-Hurler polydystrophy, Pycnodysostosis, Sandhoff disease, Sanfilippo syndrome A/B/C/D, Scheie syndrome, Schindler disease, Sialidosis types I/II, Sialuria, Salla disease, Sly syndrome, Tay-Sachs disease, Vogt-Spielmeyer disease, Wolman disease
- Location: North America
The combined and integrated efforts of the Lysosomal Disease Network will focus limited resources toward creating a network of centers with expertise in one or more of these diseases in order to solve major challenges in diagnosis, disease management, and therapy. Solutions to these problems will have direct impact on patients suffering from lysosomal diseases and important implications for medical practice.
The network is studying the following diseases:
alpha-Mannosidosis types I / II
Batten disease, late infantile
Galactosialidosis types I / II
GM1-Gangliosidosis types I/II/III
Mucolipidosis type IV
Mucopolysaccharidosis type IX
Multiple sulfatase deficiency
Sanfilippo syndrome A
Sanfilippo syndrome B
Sanfilippo syndrome C
Sanfilippo syndrome D
Sialidosis types I / II
Sialuria, Salla disease
Structure & Governance
This consortium is part of the Rare Diseases Clinical Research Network (RDCRN), an initiative of the Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS). The RDCRN is designed to advance medical research on rare diseases by providing support for clinical studies and facilitating collaboration, study enrollment, and data sharing. Through the RDCRN consortia, physician scientists and their multidisciplinary teams work together with patient advocacy groups to study more than 200 rare diseases at sites across the nation.
Funding and scientific oversight for the RDCRN are provided by NCATS and 10 NIH components: Eunice Kennedy Shriver National Institute of Child Health and Human Development; National Cancer Institute; the National Heart, Lung and Blood Institute; National Institute of Allergy and Infectious Diseases; the National Institute of Arthritis and Musculoskeletal and Skin Diseases; National Institute of Dental and Craniofacial Research; National Institute of Diabetes and Digestive and Kidney Diseases; National Institute of Mental Health; National Institute of Neurological Disorders and Stroke; and Office of the Director. In addition, patient advocacy groups provide funds for many of the projects.
Points of Contact
Chester B. Whitley, Ph.D., M.D.
Principal Investigator, Lysosomal Disease Network
University of Minnesota Twin Cities
MMC 446 Mayo
420 Delaware Street, SE
Minneapolis, MN 55455
David CC Erickson
Informatics Director, Lysosomal Disease Network
James C. Cloyd, Pharm.D.
Co-Principal Investigator, Lysosomal Disease Network
Brenda Diethelm-Okita, M.P.A.
Program Manager, Lysosomal Disease Network
Evelyn S. Redtree, M.S.
Writer & Editor, Indications
The newsletter of the Lysosomal Disease Network
Sponsors & Partners
Albert Einstein College of Medicine, Children’s Hospital at Montefiore
Baylor Research Institute and Baylor College of Medicine
Emory University School of Medicine
Hospital for Suck Children
Joan and Sanford Weill Medical College of Cornell University
Los Angeles Biomedical Research Institute at Harbor- UCLA Medical Center
Mayo Clinic, Rochester
New York University School of Medicine
State University of New York
University of California at San Francisco
University of Minnesota
University of Washington