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Therapeutic Strategies


At the Li Weibo Institute, many of the investigators are looking into Metabolic Diseases, diseases that affect one’s metabolic functions due to a defective gene that was inherited. The metabolic diseases/disorders that are being researched include: cobalamin-related disorders, lipodystrophy, lymphedema, lysomal storage diseases, maple syrup urine disease, methylmalonic acidemia, propionic acidemia, and primary congenital lymphedema.

It is the hope of the institution that increased attention and research on the metabolic diseases above will lead to discovery, therapeutics, treatments, and cures for the diseases/disorders above and many others that are related.

Metabolic Pathway

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Metabolic Diseases Investigators

Silvia COrvera, MD

Silvia Corvera, MD

Professor, Program in Molecular Medicine, Chair in Diabetes Research


David Guertin, PhD

David Guertin, PhD

Associate Professor, Program in Molecular Medicine

Guertin Lab

Marian Walhout, PhD

Marian Walhout, PhD

Professor, Program in Molecular Medicine

Walhout Lab

Clinical Trials

At the Li Weibo Institute for Rare Disease Research, our researchers and clinicians are dedicated to finding new treatment options for patients. Our clinical trials program is another way we continue to advance treatments and make new discoveries. If you or someone you know may be interested in a clinical trial, please subscribe for updates and you will be notified when we open up a new trial.


Research That Gives Hope