Angels for Alyssa

Research

A research team from the National Institutes of Health (NIH), led by Charles Venditti, M.D., Ph. D. Genetics, is conducting extensive research on mut 0 MMA. Patients, predominantly children, from all over the world participate in the research studies.

Julien Sénac and Dr. Venditti in the research lab, 2011

Visit www.genome.gov/27529399 for more information about the clinical studies.

The Clinical Center at the NIH in Bethesda, Maryland is the nation's largest hospital devoted entirely to clinical research. It is a national resource that makes it possible to rapidly translate scientific observations and laboratory discoveries into new approaches for diagnosing, treating, and preventing disease.

At NIH, Dr. Venditti and his team have developed models of mice and other primitive organisms affected with MMA for manipulation in the laboratory.

The primary goal is to develop improved treatments and ultimately a cure for this metabolic disorder through gene, stem cell and small molecule drug therapies.

For a list of published work related to the studies, visit www.genome.gov/19016902

Jennifer Sloan PhD, MS, Alyssa (Mut 0 MMA), Charles Venditti, M.D., Ph. D. Genetics and Kayleigh (Mut 0 MMA) during a 2011 visit to NIH for the clinical studies.

Angels for Alyssa entered into a donor agreement directly with the National Genome Research Institute on January 1, 2010 and has been responsible for the administration of the MMA Research Fund since that time. Previously, the Organic Acidemia Association administered the MMA Research Fund.

Visit www.mmaresearch.com to learn more about the research efforts at NIH.

Alyssa with

Dr. Venditti,

Jennifer Sloan PhD, MS

and her friend, Stephanie,

who also suffers

from MMA.