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:

The Bray Family

The Bray Family

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.

After 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

Watch the video shown during NORD’s 2012 “Partners in Progress Celebration”, telling the story of one family who was impacted by Anascorp®:

 


Nashville’s Milton Ellis is Honoree of National Organization for Rare Disorders June 2000

Nashville, Tennessee – June 2000 Milton Ellis, President/CEO of Nashville-based Rare Disease Therapeutics, Inc., was honored for his efforts to help individuals afflicted with rare disorders in this country as well as internationally. This was announced in June of 2000 by Abbey Meyers, then President of the National Organization for Rare Disorders (NORD) in Washington, D.C.

“It’s an honor for me to present Mr. Ellis with this Humanitarian Award, “said Ms. Meyers at the time. “His efforts to help find relief for those suffering from a variety of rare disorders goes way beyond the norm. Mr. Ellis and his company, Orphan Pharmaceuticals USA, Inc. (now Rare Disease Therapeutics, Inc.), are continually searching for ways to assist patients and their families.”

Ellis was recognized specifically for the development of Orfadin®, a treatment for children with hereditary tyrosinemia type 1 (formerly distributed by Rare Disease Therapeutics, now distributed by Swedish Orphan Biovitrum). This is a life-threatening genetic metabolic disease affecting infants and children. Assisting Ms. Meyers in presenting the award was Danielle Barckett of Little Falls, New Jersey, a patient with the disease, who was, at the time, in excellent health and leading the normal life of a four-year-old.

Milton Ellis accepting NORD Humanitarian Award
Pictured from left to right: Abbey Meyers, NORD President Emeritus; Milton Ellis, Rare Disease Therapeutics; Danielle Barckett; Pam Barckett

 

Rare Disease Therapeutics, Inc. specializes in pharmaceutical products to treat orphan diseases, which are considered to be diseases afflicting less than 200,000 Americans. One out of every 12 people has a rare disorder. In 1983, Congress enacted the Orphan Drug Act to give companies financial incentives and regulatory assistance in developing products to treat rare diseases. Such products are designated as “orphan products” by the FDA’s Office of Orphan Product Development.

Rare Disease Therapeutics, Inc., founded in 1991, is a profitable, privately-held company and is a leader in the orphan pharmaceutical marketplace.

 

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