Guardians of the last mile: Ensuring smooth delivery and a seamless patient experience

Patients are at the heart of Sarepta's mission to engineer precision genetic medicines, challenge convention and drag tomorrow into today. We understand that our commitment to individuals impacted by rare genetic disease isn’t limited to research and development. In this article, we introduce a few of the Sarepta team members who play a pivotal role in ensuring a new therapy for Duchenne muscular dystrophy reaches patients in a timely manner. From exhaustive attention to detail and an all-hands-on-deck mindset, to late-night calls and in-person hugs with grateful families, their dedication and teamwork highlight the essence of Sarepta's patient-first culture.

On a recent summer morning, Angie Gladieux was pacing the loading dock of a children's hospital in the Washington, DC, area. Gladieux, associate director of Market Access and Reimbursement at Sarepta, was awaiting the arrival of an important delivery: the dose of a new therapy for Duchenne muscular dystrophy. It was the first time the recently approved therapy would be administered outside of a clinical trial. Gladieux was there to make sure the shipment made it safely to its destination and the hospital staff was comfortable with the storage and administration details. 

The shipment was expected around 3 pm. Gladieux was at the loading dock by 9:45 am. “I didn’t sleep much the night before,” she admits. 

That same day, Mary Kate Mammen, a senior case manager with Sarepta’s SareptAssist Patient Services team, left Boston on a California-bound plane. For the past few weeks, Mammen had been supporting a family that was trying to gain access to the new Duchenne therapy. She was in constant contact with the family – sometimes dozens of texts in a single day – guiding them through insurance and care-related questions and challenges. When the authorization came and the dose was scheduled, Mammen wanted to be there to see the final stages of the cumulative effort. She also wanted to greet in person and hug the family with whom she had spent so many hours on the phone.  

Mammen and Gladieux were proud to play a role in bringing this therapy to patients – viewing their contributions as the final leg of a very long journey. From early research and discovery in the lab, through clinical trials and the development process, to regulatory review and approval, manufacturing, quality assurance, distribution and patient support, to a loading dock in Washington, DC, and a cross-country plane ride. 

“I’m just one piece of the puzzle,” Gladieux said. “There are so many of us who played a part in getting us to that day. It was a tremendous honor to be a part of it.”

While Gladieux was waiting for that special delivery in Washington, DC, Kara Clawson, director of Market Access and Reimbursement, was focused on ensuring a northern California hospital was prepared to administer the therapy to its first patient. This entailed meticulous coordination to navigate the therapy's unique cold-storage requirements and individualized dosing for patients. Clawson and her cross-functional colleagues also worked closely with the hospital staff, provided training on product handling, dose preparation and more. They even conducted mock deliveries and dress rehearsals to preemptively address any concerns.

But it wasn’t all planning and cold-storage details; Sarepta team members were determined to ensure that insurance-related delays did not hinder patients' access to therapies.

Mammen's role in patient support was equally crucial. Operating as a lifeline for patient families, she offered personalized assistance and addressed queries about insurance, treatment logistics, and community resources. Her involvement with the California family was particularly focused: He was set to receive treatment just before his sixth birthday – a pivotal deadline in terms of eligibility. The collective effort of Mammen, Clawson and other Sarepta colleagues ensured that obstacles were overcome and coverage was secured.

“This whole process has been remarkable, especially the immense effort and dedication shown by so many people across Sarepta. They truly stepped up and demonstrated that they were fighting for this patient,” Mammen said. “It’s the single-best example of teamwork on a cross functional level I could ever imagine. It certainly has been one of the most impactful and meaningful experiences of my career – and definitely the one with the highest stakes. I’m so thankful to have been a part of it.”

Meeting the California family in person after their son's treatment was a poignant moment for Mammen. “I saw his mom squinting at my name tag, and I just said, ‘it’s me, Mary Kate.’ Then she put her hands over her face and just started shaking, crying. I started crying. The dad started crying. It was such a happy moment,” she recalled.

The encounter affirmed that though her work can be intense and sometimes heartbreaking, she is fully committed to the families she serves and motivated by the difference she can make in patients’ lives. “It just made me think, yeah, that that's why I get up and do it all over again tomorrow.”

For Gladieux, it was only after she was headed back home after the delivery and dosing that she finally allowed herself to exhale. “It was just a tremendous moment and one that I'll never forget. Ever,” she said. 

But almost immediately, she turned her attention back to work and what comes next. “While we cherish the moment, now's the time to take the next step forward because we have more to do,” she said. “We have to continue to work with urgency and with action, always keeping those next patients first and foremost in every single thing that we do every day. They’re at the center and focus of everything that we do.”