Northwestern students will pack the Dance Marathon tent Friday to spend 30 hours dancing for Team Joseph as the primary beneficiary petitions for approval for one of the first promising treatments for Duchenne muscular dystrophy.
Marissa Penrod, Team Joseph’s founder, said DM has both increased awareness about Duchenne, the most common degenerative genetic muscle disease, and raised funds for the organization. The money raised has the potential to accelerate research on the disease, which could result in the approval of the first treatment for Duchenne, Penrod said.
“We’re at a tipping point with Duchenne where we are so close to having the treatment that this will either be the last generation to die or the first generation to survive Duchenne,” she said. “What Northwestern is doing with DM is literally life-changing, and the hope is that it’s life-saving.”
Scientists and researchers have developed a therapy targeting specific gene mutations with the potential to treat 13 percent of those diagnosed with Duchenne. This treatment has shown to be safe and effective with no recorded side effects, but hasn’t yet been approved by the Food and Drug Administration, Penrod said.
An act passed by Congress in 2012, known as the FDA Safety and Innovation Act, gives the FDA the right to approve therapies at an accelerated rate for rare diseases with “unmet medical need” — a category that includes Duchenne.
Penrod said the treatment in question has been tested and received support from multiple scientists, yet the FDA has delayed giving its official approval.
“So the big question right now is why, why is the FDA not granting accelerated approval to this when it has shown to be efficacious and safe?” Penrod said. “We just don’t have time to wait, and I think above all else there needs to be an acknowledgement that our kids are not their science experiment.”
DM’s executive board selected Team Joseph in May out of a pool of more than 75 applicants. DM spokeswoman Summer Delaney said when choosing this year’s primary beneficiary, Team Joseph was a compelling option.
“I think this year what we really loved about Team Joseph is that we had a chance to make substantial impact,” the Medill junior said. “I think NUDM is really going to help find a cure or treatment for Duchenne, but we’re just beginning.”
Team Joseph is part of a larger network of Duchenne foundations that created The Race to Yes, a website with information on the disease and a link to a recent White House petition. Penrod said creators hope to get 100,000 signatures within 30 days to guarantee the government looks into the issue.
Penrod said she hopes the process will set a precedent for the future, emphasizing the importance of rapidly approving treatments for diseases like Duchenne that have a time limit.
“It’s really time that families are allowed to make the decision if they want to try it for their children,” Penrod said.
The money raised by DM will help to both fund research and initiate a family assistance program in the Chicago area that will help families pay for Duchenne-related expenses, Penrod said. She said, however, the foundation’s primary mission is to facilitate research with the potential to impact an entire generation of children.
Penrod said after the event, she is looking forward to working with Team Joseph’s scientific advisers and researchers to most effectively direct the funds to cause the greatest impact.
After months of anticipation and working with DM, Penrod said, she is excited for the weekend to arrive and appreciates the time and effort of each individual dancer. She said she is still in shock that her organization was chosen.
She said above all, she will be “sad when it’s over.”
“The last nine months, I felt like I’ve had this and I have — we have — this entire army of supporters helping to drive our mission in making a difference for what Team Joseph does,” Penrod said. “That has been an incredible gift.”