Scientists are investigating treatments for individuals with disease-causing mutations in the MEF2C gene. These treatments could be groundbreaking since there are currently no cures for this condition, which causes lifelong mental and physical disabilities.
The next step is to conduct a detailed study of our patient population. This study will help us to better understand our patients and to prepare for a clinical trial. It will be used to evaluate the effectiveness of treatments in a clinical trial, which the FDA requires for approval.
The US MEF2C Foundation is collaborating with Wendy Chung, MD, PhD, clinical and molecular geneticist and Chief of the Department of Pediatrics at Boston Children’s Hospital and Harvard Medical School, a leader in the rare disease arena.
The US MEF2C Foundation is raising funds for our project with the lab of Dr. Wendy Chung at Boston Children’s Hospital. These funds will be used to study our patients and gather important information about their condition. Your donation will fund evaluations of our patients and the creation of a comprehensive assessment record, called a natural history. This patient based research will form the basis for measuring how well the treatments work.
The ultimate goal is to be ready for clinical trials as soon as possible and to accelerate the speed at which treatments are available to patients.
The US MEF2C Foundation needs your support to reach this goal. All contributions will help to accelerate treatments and a cure for our patients.