What We Do

Impact

In our first 8 years of fundraising prior to and after the organization's formation, we have raised over half a million dollars to put toward the fight to end Duchenne. Your contributions have been pooled with other like organizations and donated to fund significant research efforts that ultimately were translated into treatment options today. By supporting the Alex Rezkalla Foundation, you are giving children living with Duchenne the chance to grow up

Advocacy

In order to find a solution to a problem, you first have understand what you are trying to solve for. Duchenne affects approximately 1 in every 5,000 boys born worldwide. There are an estimated 15,000 patients living with Duchenne in the United States and 300,000 across the world. There are no cures today.

Often times families are unaware of the disease until they receive a diagnosis and it is then that friends, extended family, and that patient’s support system often first hear the word Duchenne. Our advocacy comes in a variety of forms. It is always intentional, however, and aimed at generating awareness and educating others. We believe telling our very personal story - the story of a boy and his dog trying to end Duchenne Muscular Dystrophy - is the key to our success and the outcome of that work will deliver funding to support critical research.

Our advocacy work comes in many forms and began months after diagnosis when Dawn attended the FDA AdComm hearing to show support for the treatment Alex is receiving weekly today, She later testified at an external review hearing to assess the benefit of Alex’s treatment. Advocating is often times educating people about real life. Dawn has participated in panel discussions about brain impact and lung health for virtual Duchenne national conferences. She filmed an interview used to describe the patient and care giver experience in Duchenne brain health for continuing education credit. Dawn was a guest on Parent Project Muscular Dystrophy’s Living Duchenne podcast to discuss topics that impact Duchenne families on a daily basis. Other times advocacy takes the shape of influencing others and asking for support on a national level, which Dawn does in Washington D.C., as part of Parent Project Muscular Dystrophy, a national Duchenne organization for which Dawn serves on the Board of Directors.

Justin is a volunteer at Nationwide Children's Hospital for the Connecting Families programs, making himself available to talk to newly diagnosed families and help them along their journey. You can always find Justin and Dawn educating others and telling their story throughout the community at local events, business networking meetings, and going into the schools Alex attends, pointing out accessibility needs and accommodates for learning. Everyone becomes an advocate when diagnosed with Duchenne.

Research

Duchenne Muscular Dystrophy has been a bit of an enigma for some time in the medical field. Although the diagnosis was well established by the 1970s, it was not until 1987 that the cause, an imperfection on the dystrophin gene, was discovered. Since that time, scientific researchers have been working to bring forward targeted approaches to improve muscle, heart, lungs, and bone health. However, treating all of the impacts of the largest gene in the body is complex and it is only in the last decade that promising research has made its way to clinical trials in humans.

Treatment options and clinical trials do not happen without participants willing to make sacrifices and take on risk in order to move the science forward. Alex was afforded a treatment option shortly after diagnosis. That option, however, came in the form of participating in a clinical trial. We strongly believe in paying it forward and after weighing the data we were given and consulting our medical team, we chose to join the fight by entering the trial. That treatment later was approved by the FDA and is now commercially available as a means to slow the progression of Duchenne. It is not a cure.

In the last five years, several other targeted treatments have been approved and more are in the making. Your support has enabled us to fund specific research focused on cardiac muscle, dystrophin restoration, immune suppression, and re-dosing with gene therapies. Dystrophin restoration through delivering a micro version of the dystrophin gene to patients was most recently approved by the FDA. Science is advancing.

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