all patients in the patient stories

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 AskBio’s founders, the answers are in our grasp.

Lisa’s story

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.

“…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."

Read Lisa’s story
Dakota’s story

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.

“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.”

Deana (Dakota’s mom)
Read Dakota’s story
Sammy’s story

Sammy’s story

Sammy is barely entering her teens and the only life she has known is one with limb-girdle muscular dystrophy type 2I/R9.

“I'm not afraid of much. Sometimes I'm afraid of limb-girdle, but most of the time I'm not.”

Read Sammy’s story
Princeton’s story

Princeton’s story

Princeton has been living with Pompe disease for over 20 years, and his ability to walk and take care of himself has been stolen from him.

“My ultimate wish is to help people...I've learned since becoming immobile, when you need help, everything is either super-expensive or you don't get it at all.”

Read Princeton’s story