Clinical and Basic Investigations of MMA and related disorders

Clinical and Basic Investigations of MMA and related disorders

The National Institutes of Health (NIH) sponsored research study named Clinical and Basic Investigations of Methylmalonic Acidemia (MMA) will evaluate patients with MMA and related disorders to learn more about the genetic causes of the various types of these inherited metabolic disorders and the medical complications associated with them. Dr. Venditti and his research team will soon begin clinical trials to test several new genomic treatments. We are fortunate to have Dr. Irini Manoli as a speaker at the upcoming International Homocystinurias Patient Expert Meeting in Rome. Dr. Irini Manoli is part of the team at NIH featured in a recent video. Dr. Irini will speak to the progress in CblC treatment. We are also fortunate to have Ms Luana Brito, also featured in the video, to speak to the news and progress of cblC Brazil.

See more about the research happening at the NIH below: