![]() DRDMG also regulates Investigational New Drug Applications (INDs), New Drug Applications (NDAs), and Biologics License Applications (BLAs) for drugs and biologics intended for the prevention and treatment of rare inborn errors of metabolism including: I just want to say, ‘Thank you for my daughter’s new life.The Division of Rare Diseases and Medical Genetics (DRDMG) serves as a hub for rare disease drug development across the Office of New Drugs by coordinating rare disease education, policy, research and stakeholder engagement. However, in 2018, there were 875 pediatric organ donors.įor Cubas, there is one donor and family in particular for whom he will forever be grateful. Of those waiting for a transplant, more than 500 are between 1 and 5 years old. ![]() The week takes aim at ending the pediatric transplant waiting list and affords members of the transplant community an opportunity to reflect on the children’s lives saved.Īccording to Donate Life America, approximately 2,000 children younger than 18 are on the national transplant list. The occasion falls annually on the last week of April, which is National Donate Month. I know she doesn’t fully understand it yet.”Ĭubas and VCU Health are sharing Astrid’s transplant journey to help commemorate National Pediatric Transplant Week. When someone meets her and I tell them what she’s been through, they can’t believe it. After several weeks of recovery in CHoR’s dedicated inpatient care units, Astrid went home. The team’s countless hours of planning went into action, making the operation and a follow-up procedure a success. ![]() It was a match and Astrid would be getting her new liver. Then, in October, the team received the much-anticipated organ offer. The process included staff run-throughs of actions and processes tailored to her case. Behind the scenes, the multidisciplinary VCU Health team prepared for her care before, during and after the transplant. (Photo by Kevin Morley, University Relations)Ī number of pre-surgery tests and appointments followed for Astrid. It’s local and it’s high quality, bringing in family members as active members of our outstanding collaborative team.” Roxana Sanchez and Jorge Cubas assist their daughters, from left, Astrid Cubas-Sanchez and Roxanne Cubas-Sanchez as they call for an elevator at the Children’s Pavilion following a recent appointment. “The answer is never no, it is always yes,” said Timothy Bunchman, M.D., director of pediatric transplantation at CHoR. Hume Endowed Chair of the Division of Transplant Surgery and director of the Hume-Lee Transplant Center. We serve patients of all ages who have advanced organ disease, and are privileged to be able to offer liver and kidney transplantation to children in partnership with CHoR,” said Marlon Levy, M.D., the David M. “The Hume-Lee Transplant Center’s collaboration with the Children’s Hospital of Richmond at VCU is absolutely central to our mission. ![]() The procedure, planning and coordination would be complex to ensure the life-changing outcome for Astrid. If it’s something else, then we’re going to do it,” Cubas said. “I would do anything for my kids, and if it’s going to take a transplant, then we’re going to do it. It was an option that would offer Astrid life without the G-tube, and no more worrying about the next health crisis. There is a long-term, permanent solution to maple syrup urine disease - a liver transplant. “I couldn’t stand seeing my daughter the way she was suffering,” Cubas said. She had suffered a seizure and a stroke, and her life was full of stringent dietary restrictions. For Astrid, it meant life with a gastrostomy tube, or G-tube, to deliver nutrients directly to her stomach. Even with treatment, the metabolic condition can be accompanied by a variety of symptoms. The disease, if left untreated, can be deadly. With that peace of mind, Cubas and his wife, Roxana Sanchez, waited as a surgical team with the VCU Health Hume-Lee Transplant Center - in consultation with care partners at the Children’s Hospital of Richmond at VCU - operated on their daughter.Īstrid was suffering from a rare genetic disorder, maple syrup urine disease, named for the sweet smell of an afflicted patient’s urine. “I just had faith in the doctors,” Cubas said. Cubas said he always knew his 4-year-old daughter, Astrid, was in the best of hands. Even as he and his family rushed to VCU Medical Center. But his thoughts, prayers and faith were steadfast. It would have been OK for Jorge Cubas to be scared.
0 Comments
Leave a Reply. |
Details
AuthorWrite something about yourself. No need to be fancy, just an overview. ArchivesCategories |