A Cure is Here!

Help Give Pyper—and Others With FD—Hope for the Future

Pyper is 11 years old and has spent her entire life living with the challenges of Familial Dysautonomia (FD), an extremely rare genetic disease that affects the autonomic and sensory nervous systems.

FD can impact nearly every part of the body—including blood pressure, breathing, swallowing, vision, balance, digestion, and the ability to feel pain. It is also progressive, meaning that many of these challenges can become more difficult over time.

But today, we have more hope than ever before.

A Groundbreaking Opportunity

Pyper is currently patient #2 in a groundbreaking clinical trial at NYU studying a treatment that targets ELP1—the gene responsible for Familial Dysautonomia.

Every 100 days, Pyper travels from Los Angeles to New York, where she undergoes anesthesia and a lumbar puncture to receive the investigational medication.

For the first time, researchers aren’t simply treating the symptoms of FD. This treatment has the potential to address the disease at its source—with the hope of slowing or stopping the degeneration caused by FD and possibly allowing Pyper to regain abilities or sensations she has already lost.

For Pyper, that could mean protecting her vision, improving her balance and mobility, preserving her independence, and giving her body the chance to do things FD has taken away.

Why We’re Fundraising

Participating in this trial requires our family to travel to New York every 100 days for Pyper’s treatments. And our journey won’t necessarily end when the clinical trial does.

We are raising funds to help support the costs associated with Pyper’s continued access to this potentially life-changing treatment and the medical care surrounding it, as well as our ongoing efforts to support the fight against FD.

Pyper is only the second patient to receive this treatment. What researchers learn from her may help shape the future not only for Pyper, but for other children and families living with FD.

Follow Pypers journey on Facebook (Powering Pyper) or Instagram (@poweringpyper).