Accredo Helps Hero Access Life-Changing Gene Therapy for SMA

One Therapy, One Child: How Accredo Helped Hero Access Life-Changing Gene Therapy

A Physician, a Grandmother, an Advocate

When Rosalyn Sabe Enos, MD first held her grandson, Hero, in her arms on August 8, 2024, she felt the kind of joy that fills a room—pure, overwhelming, unforgettable. But shortly after, that joy was eclipsed by a wave of disbelief, helplessness, and heartbreak. Hero was diagnosed during routine newborn screening with spinal muscular atrophy (SMA)—a rare and devastating condition that upended everything her family thought they knew about the future.

“Even as a physician, I was unfamiliar with SMA,” Dr. Enos recalls. “Googling it was the worst feeling in the world—seeing the devastating impacts of the disease laid out in front of me. I knew I had to fill in the knowledge gaps quickly and ask the right questions to advocate for my family.”

Her instinct to advocate wasn’t new—it was rooted in her own experience. Dr. Enos’s journey wasn’t just clinical—it was deeply personal.

Reflecting on her path, Dr. Enos shares, “I went to medical school to advocate for my family. As an 18-year-old single mom, the medical community didn’t treat me well when my son needed multiple surgeries. That experience shaped my path. Now, that same drive to protect and support my loved ones includes my grandchildren.”

Born and raised in Oahu, Hawaii, she raised two children as a single mother while attending college and medical school. She built a career in pathology, working as a partner of a successful private pathology group in Hawaii, and later joined a specialty gastrointestinal pathology practice.

But when Hero was diagnosed, she became something more: a caregiver, a researcher, and a fierce advocate navigating the complex world of gene therapy.

A Glimmer of Hope

For babies diagnosed with SMA, the prognosis was often devastating. Survival was uncertain, and for those who lived, life was severely limited—no ability to walk, run, or play.

Amid this uncertainty, the family learned about a potential treatment: ZOLGENSMA®, a gene therapy that could change everything for Hero. It offered more than hope—it offered the possibility of a future not defined by SMA. But accessing it was anything but simple. Timing was critical. Every single day mattered because the therapy was weight-based, and Hero’s age and development were key factors.

Just when the path forward seemed most uncertain, Accredo entered the picture.

“We didn’t know much about specialty pharmacies,” Dr. Enos admits. “But once we connected with Accredo, everything changed.”

Everything changed because the family was no longer navigating the complex treatment journey alone. Accredo stepped in with deep expertise, guiding them through insurance approvals, coordinating with the care team, and ensuring timely access to the life-saving gene therapy. The support was immediate and personal—every question answered; every unique barrier addressed. For Hero, it meant the difference between waiting and receiving. For his family, it meant hope backed by action.

Corrie Ball, Accredo’s Gene Therapy Program Manager, became a lifeline. She helped coordinate care, navigate logistics, and accelerate access to Hero’s treatment—even as a potential nursing strike threatened the hospital’s ability to administer the multi-million-dollar therapy. Her proactive coordination ensured that nothing stood in the way of Hero receiving the therapy on time. Her role exemplified the kind of personalized, high-stakes support gene therapy patients and families need.

Thanks to Accredo’s support, Hero didn’t just receive a therapy—he received a chance at life without the complications associated with SMA.

A Network of Care

Hero’s journey wasn’t just about one drug or one doctor—it was uniquely his. Every step required a personalized approach, tailored to his medical needs, family circumstances, and the urgency of treatment.

Accredo rose to the occasion through its small, hyper-focused GeneAXS team—experts in gene therapy who deliver deeply personalized support. This tight-knit group of gene therapy experts became an extension of Hero’s care circle, working seamlessly with his providers to navigate approvals, coordinate logistics, and ensure timely access to therapy.

The program also screens for social determinants of health (SDoH) and offers wraparound services that go beyond medication—supporting families holistically through high-stakes moments.

“Every person on our team had a role in Hero’s care,” says Corrie Ball, Accredo’s Gene Therapy Program Manager. “We treat each patient as an individual—not a number. One therapy is one therapy, and one patient journey is one patient journey.”

A Year of Progress

On September 17, 2024, Hero received his gene therapy infusion. Today, he’s thriving—hitting, and even exceeding, his developmental milestones. He’s walking. He’s laughing. And he’s living proof of what’s possible when care is personalized, timely, and compassionate.

These milestones wouldn’t have been possible without the therapy. Hero’s progress is a testament to the power of timely intervention, expert coordination, and unwavering support across the healthcare ecosystem.

“We’re focused on helping him maximize the outcomes of his therapy,” Dr. Enos shares. “He’s doing physical therapy, and we’re all committed to helping him continue to thrive. The therapy was a huge part of why he’s where he is today—but it’s just the beginning.”

A Message to the Healthcare Community

As she reflects on the journey, Dr. Enos offers a message to those working across the health care ecosystem:

“Keep Hero in mind when you do what you do every day. You are making a difference.”

Turning Hope into Reality

Hero’s story is a reminder that behind every therapy is a family holding onto hope. And with the right partnerships, that hope can become reality. In fact, Hero became the first patient in Hawaii to receive ZOLGENSMA® gene therapy for SMA—a milestone made possible through coordinated care and unwavering advocacy.

Read more about Hawaii’s first ZOLGENSMA® treatment on Accredo Insights.

Accredo’s gene therapy support model—including Therapy Link—is built to serve the entire healthcare ecosystem. From prescribers and manufacturers to caregivers and patients, Accredo delivers the coordination, clinical support, and wraparound services needed to make complex therapies possible.

Learn how Accredo and Evernorth Health Services’ Therapy Link are transforming access to gene therapy across the healthcare continuum.