Tushar Puthran

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 will help us to better understand our patients and to prepare for a clinical trial. This information 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 Boston Children’s Hospital and Harvard Medical School, a leader in the rare disease arena, to better understand MEF2C patient disease.

The US MEF2C Foundation is raising funds for projects with Boston Children’s Hospital / Harvard Medical School. These funds will be used to study our patients and gather important information about this disease. This research will form the basis for measuring how well 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.