PROJECT/ALIVE families featured at international symposium on rare disease in San Diego

 
 
Jenn, Sebastian and Mario Estevez will be in San Diego this week.
Jenn, Sebastian and Mario Estevez will be in San Diego this week.
 
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Hunter Syndrome

Industry:
Medical

Location:
Nashville - Tennessee - US

Subject:
Events

NASHVILLE, Tenn. - July 30, 2018 - PRLog -- Project Alive families from Nashville and South Florida will travel to San Diego, California, Aug. 2-4 to learn from and connect with other families from across the globe who are touched by forms of Mucopolysaccharidosis, one being Hunter Syndrome.

Project Alive (https://projectalive.org/) President Melissa Hogan will present on "Managing the Complex Journey for MPS II," at the 15th International Symposium on MPS & Related Diseases (https://mps2018.com/) on Aug. 4. Her son Case was diagnosed with Hunter Syndrome, also known as MPS II, as a toddler. Since his diagnosis, Hogan has become a recognized expert on the disease. She was recently nominated to serve on the Clinical Trials Transformation Initiative (CTTI) and U.S. Food and Drug Administration (FDA) Patient Engagement Collaborative (PEC), as a part of which, she will meet with FDA officials several times a year to discuss the best ways for patients to take part in regulatory discussions about medical products. She is also a 2018 RARE Champion of Hope award nominee, recognized by rare disease nonprofit Global Genes.

Mario Estevez, Vice President of Project Alive, and his wife Jennifer will also be attending with their sons Omar and Sebastian, who also has Hunter Syndrome. The Estevez family's South Florida community played an integral role in helping Project Alive raise over $1 million in 2017. Many Broward County public and private schools, along with local businesses, hosted fundraising events to support Project Alive. They joined forces with a group of students at Florida Gulf Coast University to raise money for Project Alive through the students' own Hunting for a Cure organization. Finally, the Estevez family began working with Petland in Pembroke Pines, Florida, to support Hunter Syndrome families with therapy dogs.

The Hogan and Estevez families, along with many others, make up Project Alive. Since 2014, Project Alive has raised more than $2 million for research and the development of gene therapy clinical trial for Hunter Syndrome.

Project Alive is about $650,000 away from being able to fund this gene therapy clinical trial at Nationwide Children's Hospital in Columbus, Ohio. The organization has already raised $1.8 million to produce the drug to be used in the trial for MPS II patients, and to support an investigational new drug (IND) application that was received in December 2017. The trial is the culmination of two decades of gene therapy research.

"The real tragedy of our story is not that our son has been diagnosed with a deadly disease, but that researchers believe they have developed a cure but lack the funding to save him," Jenn Estevez said, adding that their mission is to continue to help Project Alive fund a cure for Hunter Syndrome.

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Barbara Esteves-Moore
***@tworoadscommunications.com
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Tags:Hunter Syndrome
Industry:Medical
Location:Nashville - Tennessee - United States
Subject:Events
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