Kurt Harzke’s son Jacob was diagnosed with Duchenne Muscular Dystrophy (DMD) when he was three years old. He’s 13 now, a smart, ambitious teen, who suffers from a disease that currently has no cure. Last month, on Rare Disease Day, February 29, Kurt and Jacob spoke to a group from the Muscular Dystrophy Association (MDA) and employees from Northbrook, IL-based Marathon Pharmaceuticals, LLC, at the company’s headquarters.
Marathon is a biopharmaceutical company focused solely on developing treatments for rare diseases, including DMD. In a few months, Marathon expects to submit a new drug application to the FDA for deflazacort, which, in clinical trials, has shown the potential to slow muscle degeneration for patients with DMD. Deflazacort is an investigational therapy in the U.S. and has not been approved by the FDA. Because there are currently no approved treatments in the US for DMD, many in the rare disease community are looking at Marathon’s submission as an important milestone.
To bring the observance of Rare Disease Day to a community level, the MDA’s Chicago office partnered with Marathon Pharmaceuticals to host Kurt and Jacob at an event that featured select pieces from the Muscular Dystrophy Association Art Collection, created by kids and young adults with DMD. Marathon will display these touching pieces of art at their corporate offices until April 30.
Marathon employees were moved and impressed by the talent and imagination of the artists affected by DMD. A number of employees commented on how the artwork reflects the artists’ view of the world, and that DMD may affect the body, but not the creativity, hopes and dreams of these brave boys and young men. Several Marathon employees donated to receive artwork by local artists affected by muscular dystrophies at the 2015 Muscle Gala in Chicago. These pieces are currently hanging in their personal offices. The overall DMD Collection further inspires the company that our daily roles will potentially impact families affected by DMD.
During the event, Kurt spoke about the realities of Jacob’s diagnosis, its impact on the family and the challenges the DMD community faces. “For parents living with [a DMD diagnosis], every day is rare disease day. We don’t have the choice to not be aware,” he said.
“I’m not here to talk about a disease, I’m here to talk about a family. While the disease is central to that – it touches everything in our lives – it is not what I see when I look at Jacob,” said Kurt. “I see a child with promise, who is very bright and articulate; [someone who] has great and creative ideas. I see a kind and caring child who is inspiring to me and his mother.”
With Jacob by his side, Kurt spoke about the difficulties of tackling DMD, as well as the progress made during the 10 years since Jacob was diagnosed. He highlighted the expanded resources in the Chicago area: the Harzke family used to travel five hours to Cincinnati for Jacob to be treated by a DMD specialist. Now, they have options in Chicago thanks to four clinics at local hospitals, set up with the support of the MDA.
The promise of how far things have come, and the knowledge of the long road ahead, was a theme of discussion throughout the event. So was the hope that things will continue to get better. The Harzkes remain determined and optimistic, thanks to the collaboration between patient, advocacy, and industry partners to better understand DMD, and to spread the word in communities across the country.
“This event is what it means to raise awareness on Rare Disease Day. It’s discussing the challenges of those with Duchenne Muscular Dystrophy and other muscular dystrophy diseases. It’s having someone explain the realities of what patients and families are dealing with and generally bringing awareness to the disease and its struggles,” said Megan Burns, associate director of clinical and family services for the Illinois and Wisconsin chapters of the MDA.
Kurt Harzke added, “To have partners like Marathon and the MDA willing to reach out, saying ‘we understand and we’re here to help’… that is an amazing thing.”
About Deflazacort
Deflazacort is a glucocorticoid with anti-inflammatory and immunosuppressant properties. Based on published clinical studies, it appears that deflazacort may be an important new treatment option for patients with DMD. Side effects reported to date include cushingoid appearance, hirsutism, weight gain, erythema, nasopharyngitis, irritability and cataract formation.
Deflazacort is currently not approved in the U.S. but is available outside the U.S. for many approved uses not including DMD. The FDA previously granted Fast Track status, Orphan Drug designation and Rare Pediatric Disease designation for deflazacort for the treatment of patients with DMD.
About ACCESS DMDTM
Through Marathon’s ACCESS DMDTM program, deflazacort is now being made available to qualified patients, at no cost, through a growing network of medical research centers across the country.
Medical centers participating in ACCESS DMDTM are up and running across the country and the number is expected to increase rapidly. The medical centers currently approved are in Little Rock, AR; Chicago, IL; Kansas City, KS; Boston, MA; Charlotte, NC; Hershey, PA; Memphis, TN; Houston, TX; Norfolk, VA and Seattle, WA.
Patients, families and physicians can learn more about ACCESS DMD™, including a list of clinical sites participating in the program, by visiting http://www.AccessDMD.com or calling 1-844-800-4DMD (4363). Further information is available at ClinicalTrials.gov.
For more information the Muscular Dystrophy Association and the local chapter: https://www.mda.org/
For more information on Marathon Pharmaceuticals and its development of treatments for rare neurological and movement disorders: http://marathonpharma.com/
