In 2006, my son was diagnosed with an ultra-rare metabolic disorder, Glutaric Aciduria Type 2/MADD. It was picked up on his newborn screening and he became Mt Sinai (NYC) first case. Our lives changed dramatically as we grappled with this new reality.

In 2009, our lives took another turn when my son caught RSV.  He was hospitalized and had to be put on life support because his lungs collapsed.  During that stay we were asked if we'd be willing to try an investigational medication used in other countries to successfully treat GA2, and we said yes.  His Dr's worked with the FDA and anyone else they needed to in order to get that medication, Sodium DL-3-Hydroxybutyrate, here for him and he has been taking it ever since. This medication is a ketone that people with my son's diagnosis cannot make on their own, and it turned out to be nothing short of a miracle. It has not only improved my son's quality of life but also gave us hope for a bright future.

Recently it was discovered that Sodium DL-3-Hydroxybutyrate treats more than just GA2; it can help many Fatty Acid Oxidation Disorder patients as well. However, despite its potential benefits to many suffering from these rare disorders, there has yet to be a clinical trial conducted here in the United States.

We are calling on health authorities and pharmaceutical companies to initiate a clinical trial for this orphan drug here in the USA. This is not just about my family or even about those currently diagnosed with these rare disorders - this is about future generations who could benefit from this treatment.

Let's give them hope and better options for treatment by initiating a clinical trial for Sodium DL-3-Hydroxybutyrate in the USA today! Everyone should have the right to try and the right to a happy, healthy, long life.  Please sign our petition to make this happen!