Fighting For A Cure: Clara’s Life With GM1

Clara Bragg kid living with GM1
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Two-and-a-half-year-old Clara Bragg is a bright and energetic toddler, feisty and friendly, a shining star who strives to keep up with her big brother and wins over everyone she meets.

But she’s running out of time—and unless something changes soon, her precious life will be cut short. When Clara was born, nothing seemed amiss. It wasn’t until shortly after her first birthday that she began to plateau developmentally. Her loving parents watched with concern as she lost the few words she spoke and struggled to walk independently.

A year later they got their devastating answer: Clara has a terminal neurological condition called Late Infantile GM1 Gangliosidosis. The prognosis is grim. The disease will slowly rob her of her abilities and ultimately cause seizures, blindness, deafness, and the inability to eat and move.

If you’ve never heard of this disease, you’re not alone. Only two children in Alabama have been diagnosed with GM1. Sadly, the other child (Porter Heatherly, age 4) recently passed away.

Porters 4th Birthday
The Bragg and Heatherly families for ‘Porter’s 4th Birthday.’ Porter Heatherly (age 4) was the only child in Alabama, other than Clara, diagnosed with GM1. More may be found on his story on Facebook at Prayers for Porter.

Clara’s family, friends, and loved ones are on a mission. Though no treatment or cure for GMI exists currently, there’s major reason for hope: The world’s two leading GMI researchers are on the brink of a potential cure right at Auburn University.

“Animal trials have proven that this cure is effective,” says Clara’s mother, Jenny Bragg. “It is just a matter of getting it to human trial so children like Clara can be treated and their lives can be saved.”

The roadblock? Money. Though families affected by this disease and the Cure GM1 Foundation have raised significant funds, they’re short the amount necessary to fund a human gene therapy trial in 2017.

The family is asking people to donate to the Cure GM1 Foundation. If the trial is funded, their beloved daughter may go on to live a long and happy life. The alternative is unthinkable: Clara’s loved ones will watch her slowly fade away from this cruel disease.

Her parents express their gratitude to everyone who helps them get closer to their goal of getting Clara into the potentially lifesaving trial. “Thank you all for your continued support,” the family wrote recently on A Cure for Clara Facebook page. “We are so hopeful we can save Clara’s life.”

“If proven successful in treating GM1, this gene therapy will then be applied to helping cure many other childhood genetic diseases. This truly is medical history in the making.” -Jenny Bragg, Clara’s Mother

How You Can Help Save Clara

BRAGG FAMILY

This human gene trial has so many expenses: vaccine manufacturing, FDA-required vaccine toxicity testing, and a full-time nurse to help manage the trial.

Clara’s family is asking people to donate “whatever their heart and means allow” to the Cure GM1 Foundation to fund these expenses and potentially help save the lives of Clara and other children with GM1.

All donations go straight to the foundation and are 100 percent tax-deductible. They aren’t used by the family for Clara’s care.

Learn More About GM1

The name sounds like a tongue twister: GM1 Gangliosidosis. This rare disease is caused by the body’s producing an insufficient amount of the enzyme beta-galactosidase. The result is cell death—in particular, spinal cells and neurons—and what happens next is devastating. Afflicted children lose the ability to do virtually any bodily function they need to live: speak, swallow, and even move. Some become blind or deaf. The heartbreak GM1 leaves in its wake is truly staggering.

There are three classifications of GM1, and they range in severity:

  • Type 1 (Infantile) is the most common and the most severe.
  • Type 2 (Late Infantile and Juvenile) is less severe than Infantile. (Clara’s GM1 is Late Infantile.)
  • Type 3 (Late or Adult Onset) is rarer and thought to occur mostly in Japan

Auburn University Gene Therapy Research

Clara and Dr. Martin
Dr. Doug Martin, pictured with Clara.

Dr. Doug Martin is one of the leading GM1 researchers in the world. He and his team at the Scott-Ritchey Research Center at Auburn University have been researching gene therapy techniques in collaboration with UMass, Lysogene and the Tay-Sachs Gene Therapy Consortium. They have successfully extended the lives of cats in their studies, and their goal is to apply that knowledge to human trials.

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