After losing his son to a rare genetic disease, state Rep. Adam Anderson vowed to make it easier for other families to find answers.<\/p>\n
That promise became reality with the passage of House Bill 907, the Sunshine Genetics Act, which paves the way for a pilot program to help families identify serious conditions at birth through gene sequencing \u2014 at no cost. Gov. Ron DeSantis signed the bill into law in July.<\/p>\n
The bill also establishes the Florida Institute for Pediatric Rare Diseases, a research center and clinic dedicated to children\u2019s diseases at Florida State University, and the Sunshine Genetics Consortium, a collaborative effort among Florida universities, including UF, to expand research and improve access to genetic testing.\u00a0<\/p>\n
Anderson appeared at a press conference Friday in Malachowsky Hall, standing alongside a group of doctors and affected families, including UF Health President Dr. Stephen Motew.<\/p>\n
\u201cSo many of these families who have a child in this diagnostic odyssey are traveling around the country seeking answers,\u201d Anderson said after the conference. \u201cAll they want to know is, \u2018What\u2019s wrong with my baby?\u2019 And unfortunately, so many of them end up passing away before they receive any treatment at all.\u201d<\/p>\n
Anderson entered politics after his son, Andrew, died from Tay-Sachs disease at age 4.<\/p>\n
The Sunshine Genetics Act was the first bill that Anderson \u201cpersonally wrote from scratch.\u201d\u00a0<\/p>\n
\u201cThe bill started as literally bullet points on a legal pad. Some of them were literally on a napkin,\u201d Anderson said to the Alligator. \u201cIt took a while to get that into a legal format \u2014 something that\u2019s actionable, something that really could become law.\u201d<\/p>\n
Joe Dion, a father to two children with limb girdle muscular dystrophy, also spoke at the event. He described his children\u2019s condition as a muscle-wasting disease that eventually \u201crobs your ability to walk.\u201d Initially, his family struggled to find treatment options for the disease. Dion travelled to the press conference from Massachusetts.\u00a0<\/p>\n
“Finding the news that they have a genetic disease, a rare disease that\u2019s incurable, zero treatments at the time, was horrible,\u201d Dion said to The Alligator. \u201cWe could have sat there and been depressed and felt bad about ourselves, which we did at the beginning \u2026 Then, we decided to get to work.\u201d<\/p>\n
Eventually, the family began working with Dr. Barry Byrne, director of UF\u2019s Powell Gene Therapy Center, and a French company called Atamyo. They traveled across the country, and the world, looking for a hospital that would treat such a difficult case, he said.<\/p>\n
Dion said gene therapy and genetic medicine has changed the \u201ctrajectory\u201d of his children\u2019s lives. Currently, his daughter is a cheerleader, and his son is a junior lobster fisherman.\u00a0<\/p>\n
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Also in attendance were Kelly Brazzo and her daughter, Sammy, who came from Pennsylvania at the invitation of Byrne. Sammy also has limb girdle muscular dystrophy. She was diagnosed at age 2 and is now 17.<\/p>\n
\u201cPeople ask us all the time, \u2018How did you get her diagnosed so early?\u2019\u201d Kelly said. \u201cReally, I mean, we both have a bit of a medical background.\u201d<\/p>\n
Kelly worked as a speech therapist and her husband as a podiatrist before starting a nonprofit called CureLGMD2i Foundation after their daughter\u2019s diagnosis.\u00a0<\/p>\n
Sammy, who is still figuring out what she wants to do in the future, said her diagnosis explained a lot.<\/p>\n
\u201cI didn\u2019t really understand it, but I was kind of happy that it was all \u2026 just finally making sense to me,\u201d Sammy said.<\/p>\n
Sammy has recently become more involved in her parents\u2019 organization, and she gave testimony about her condition at the family\u2019s church.<\/p>\n
Sammy also recently completed her first advocacy event and shared her story with legislators in Pennsylvania.\u00a0<\/p>\n
Byrne, who is trained in the field of genetics and pediatric cardiology, said the involvement of the family in the treatment process is \u201ccritical.\u201d<\/p>\n
\u201cWe have to know what’s meaningful to the family that would represent an effective treatment,\u201d Byrne said to the Alligator.\u00a0<\/p>\n
The press conference concluded with a tour of the Intelligent Clinical Care Center in Malachowsky, UF Health Shands Hospital and Clinical and Translational Research Building.<\/p>\n
Julianna Bendeck is a contributing writer for The Alligator. Contact her at jbendeck@ufl.edu<\/p>\n
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