Rare disease Therapeutics Nashville

The Bray Family

Awards and Recognition

Rare Disease Therapeutics, Inc., has been recognized in the community as a leader in the area of rare diseases. Below are some of Rare Disease Therapeutics, Inc.’s recent achievements.

National Organization for Rare Disorders (NORD) Partners in Progress Corporate Awards – May 2012

Rare Disease Therapeutics, Inc., for Anascorp® for treatment of patients for scorpion stings.

From the Partners in Progress Gala Program:

Wayne and Dawn Bray lost their two-year-old son, Dally, to a scorpion sting in 2002. The Brays live in a small rural community in Arizona, the state in which the majority of the 20, 000 or so reported scorpion stings in the U.S. each occur.

Severe venomous scorpion stings occur most frequently in infants and children, and can cause shortness of breath, fluid in the lungs, breathing problems, excess saliva, blurred vision, slurred speech, trouble walking, and other uncoordinated muscle movements. Untreated cases can be fatal.

Dally died within hours of being stung by a scorpion, and the Brays live with the painful memory of desperately seeking help as their little boy exhibited sudden and frightening symptoms.

When their youngest son, Morgan, also suffered a scorpion sting a few years later, the family rushed him to the nearest rural hospital. From there, he was taken by helicopter to a Tucson hospital, where he was given an anti-venom that saved his life. Today, Morgan is fine.

That product, known as Anascorp®, is designed specifically for scorpion stings and provides new treatment for both children and adults. It is made from the plasma of horses immunized with scorpion venom.

The U.S. Food and Drug Administration (FDA) approved Anascorp® as a treatment for scorpion stings in August 2011. The product is manufactured in Mexico, where scorpion stings are far more common, but is licensed to Rare Disease Therapeutics, Inc., which obtained orphan designation and conducted clinical trials leading to its approval.

Milton Ellis accepting NORD Humanitarian AwardAfter learning of the FDA approval, Dawn Bray said she felt “joy and elation” that this treatment would now be available to other families going through what her family experienced with Dally. It leaves her with a feeling that her son “didn’t pass away in vain, ” she says.

The Bray family has worked with Dr. Leslie Boyer at the University of Arizona, who was the lead investigator on the clinical trials of Anascorp®, and others in that university’s “VIPER” Institute (Venom Immunochemistry, Pharmacology and Emergency Response Institute) to share their story with physicians and medical students to alert them to the dangers of venomous scorpion stings.

Rare Disease Therapeutics, Inc., has a history of commitment to the rare disease patient community. This evening, NORD salutes the company for this new treatment for children and adults experiencing severe venomous scorpion stings.

VIDEO FROM NORD 2012

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We are both curious and fearful of any new disease that we hear about. We stayed glued to the TV news if we hear of an “outbreak” of some sort. We fear as much as we investigate.

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