Duchenne muscular dystrophy is a progressive and fatal genetic disorder that affects the muscles in the limbs. There is no cure, and none are in development. (For comparison, there are experimental drugs in the works to treat epilepsy and Alzheimer’s, but those drugs do not cure either illness.)”

“Duchenne muscular dystrophy is a progressive and fatal genetic disorder that affects the muscles in the limbs. There is no cure, and none are in development. (For comparison, there are experimental drugs in the works to treat epilepsy and Alzheimer’s, but those drugs do not cure either illness.)”— Nema Faroutan, director of Children’s Hospital in Philadelphia

“The Centers for Disease Control and Prevention and their partners around the world do not have enough funding to conduct the kind of research that they need to do to understand the disease in more detail.”

“The Centers for Disease Control and Prevention and their partners around the world do not have enough funding to conduct the kind of research that they need to do to understand the disease in more detail.”— Margaret Saturveda, medical expert on DMD and consultant to the UCSF DVHM

“Fewer than 50,000 people worldwide have Duchenne muscular dystrophy, which is incurable, is caused by a rare genetic mutation, and in most cases, a woman is at an increased risk for getting it. The median life expectancy of a person with Duchenne muscular dystrophy is just 27 years old.”

“Fewer than 50,000 people worldwide have Duchenne muscular dystrophy, which is incurable, is caused by a rare genetic mutation, and in most cases, a woman is at an increased risk for getting it. The median life expectancy of a person with Duchenne muscular dystrophy is just 27 years old.”— Debra Camarand, retired Oregon State University professor of anatomy

“An incredibly severe disease will cause babies to die before they’re a year old.”

“An incredibly severe disease will cause babies to die before they’re a year old.”— Julia Bremmer, nonprofit manager for the Dian Fossey Gorilla Fund

“A devastating disease causes the pain and torment of young children as they wrestle with advanced age.”

“A devastating disease causes the pain and torment of young children as they wrestle with advanced age.”— Alfonso Pollaca, Ph.D., founding dean of UC Irvine School of Medicine and former chancellor of UC Davis

“In 2015, five years after Mark and Julia first met, Mark died. They met at Children’s Hospital of Philadelphia because Mark was 18 months old and Julia was working in the autochondroplasia department at CHOP. It was there she saw the incredible courage and passion of parents who came to find hope when nothing else would.”

“In 2015, five years after Mark and Julia first met, Mark died. They met at Children’s Hospital of Philadelphia because Mark was 18 months old and Julia was working in the autochondroplasia department at CHOP. It was there she saw the incredible courage and passion of parents who came to find hope when nothing else would.” — Nema Faroutan, director of Children’s Hospital in Philadelphia