Patient stories that drive our gene therapy clinical research
Living with genetic disease
There are no ordinary days for those who must endure the life-altering consequences of genetic disease. For some, it means a shortened life expectancy and others a life full of challenges with loss of motor function, deteriorating memory, muscle atrophy, breathing difficulty, and damage to various organs and tissues that most of us can’t imagine.
When we hear the stories of patients living with a genetic disease, they give us every reason to urgently discover new cures and treatments. Today, due the groundbreaking discoveries by gene therapy pioneers like Drs. Jude Samulski and Xiao Xiao, two of RhiaPharmaceutical’s founders, the answers are in our grasp.
Lisa’s story
Lisa can’t get up from a chair on her own, but she is not letting limb-girdle muscular dystrophy type 2I/R9 stop her from being a working mother and loving wife.
Read Lisa’s story“…the first time I was told I had a rare form of muscular dystrophy, it was a profound moment. I wanted to know more, but they said they had no more information than the diagnosis."
Dakota’s story
Dakota will soon be old enough to get his driver’s license, but he will never see a day when he takes a driving test due to early onset Pompe disease.
Read Dakota’s story“Dakota makes my life so much better seeing his positive outlook and upbeat personality—he’s a blessing for so many other families and ours.”
