When Science and Humanity Become Inseparable
A Rescue Trial Case Study
How kindness transformed the clinical trial experience for pediatric rare disease participants in a global study
"You’re not just processing payments. You’re helping us ensure they make it to the trial. And that changes everything.”
The following story reflects a conversation between Mural Health and the Head of Clinical Operations and Head of Community & Communications at Myrtelle.
The team at Myrtelle, a patient-centered gene therapy company for neurological diseases (and a customer of Mural Health),recently sat before the FDA to share their latest data from a rare disease clinical trial. But Myrtelle didn’t just present the data–they showed the people behind it.
Videos of trial participants–terminally ill children who were once unresponsive–showed them babbling, engaging, and even walking. The weight of the moment was undeniable, and the impact so profound that FDA officials were moved to tears. It revealed a truth that’s often swept under the rug in clinical research: scientific research and humanity are inseparable.
But getting to this point wasn’t easy.
Clinical trials offer hope to patients—so much so that families uproot their entire lives for it, especially for pediatric rare diseases where approved treatment is elusive. But logistical and financial barriers too often get in the way, and the outcome can be devastating.
“Genetic diseases don’t discriminate based on socioeconomic status,” said Myrtelle’s Head of Clinical Operations.“They don’t choose whether a family has millions of dollars or struggles to make ends meet. The disease is not concerned about their finances, but the support systems we put in place should be. As a clinical trial sponsor, we have to make sure participation is economically possible for every family.”
With this in mind, Myrtelle set an ambitious goal: ensuring families with medically complex needs could travel across the world to participate in their trial, overcoming logistical barriers that felt impossible. In partnering with MuralHealth, they turned their goal into reality for these pediatric patients and their families.
Videos of trial participants–terminally ill children who were once unresponsive–showed them babbling, engaging, and even walking. The weight of the moment was undeniable, and the impact so profound that FDA officials were moved to tears. It revealed a truth that’s often swept under the rug in clinical research: scientific research and humanity are inseparable.
But getting to this point wasn’t easy.
Clinical trials offer hope to patients—so much so that families uproot their entire lives for it, especially for pediatric rare diseases where approved treatment is elusive. But logistical and financial barriers too often get in the way, and the outcome can be devastating.
“Genetic diseases don’t discriminate based on socioeconomic status,” said Myrtelle’s Head of Clinical Operations.“They don’t choose whether a family has millions of dollars or struggles to make ends meet. The disease is not concerned about their finances, but the support systems we put in place should be. As a clinical trial sponsor, we have to make sure participation is economically possible for every family.”
With this in mind, Myrtelle set an ambitious goal: ensuring families with medically complex needs could travel across the world to participate in their trial, overcoming logistical barriers that felt impossible. In partnering with MuralHealth, they turned their goal into reality for these pediatric patients and their families.
"With Mural Health, I have peace of mind. I know these families are getting the support they need."
"Our previous provider couldn’t adapt quickly. It was a constant struggle."
- Myrtelle’s Head of Community & Communications
One of the hardest parts of clinical trials is data collection—you have to prove impact.This pediatric trial requires fluid samples,MRIs, gross motor tests, and a range of other assessments–all contributing to the ultimate goal of getting the drug approved so it can reach patients worldwide. But allof these assessments require in-person visits, which means travel to and from the site. Travel for a medically complex child–especially one in a wheelchair–is incredibly challenging. Some wheelchairs fold, but others don’t, which creates a whole new layer of difficulty, as airlines are not always accommodating.
Prior to Mural Health, Myrtelle was partnered with a well-known clinical research vendor for payment and travel coordination. But this vendor failed them, creating so many challenges that the trial site eventually refused to see more participants.
Prior to Mural Health, Myrtelle was partnered with a well-known clinical research vendor for payment and travel coordination. But this vendor failed them, creating so many challenges that the trial site eventually refused to see more participants.
“Reimbursements were a nightmare. Communication was difficult. Travel was extremely cumbersome. If a family missed a layover, it became a catastrophic event.”
Without real-time reimbursements or prepaid support, families are often forced to cover expenses upfront—something many simply cannot afford. This study is an example of that, where outstanding reimbursements owed to participants totaled tens of thousands of dollars.
Myrtelle’s Head of Community &Communications understands these challenges firsthand. She comes from the rare disease community she serves, giving her a more intimate relationship with the patient population.
“Families are already going through their worst nightmare. Financial and logistical barriers should never be another obstacle.Most of these kids are in diapers and have feeding tubes. There are so many extra layers to consider. With our previous provider, my inbox was constantly flooded with five-alarm fire situations—visa issues, incorrect flight bookings, missing reimbursements. Patients would reach out to the site first, but when they didn’t get a response, they’d come to me. I don’t get those emails anymore. That tells me everything.”
Myrtelle’s Head of Community &Communications understands these challenges firsthand. She comes from the rare disease community she serves, giving her a more intimate relationship with the patient population.
“Families are already going through their worst nightmare. Financial and logistical barriers should never be another obstacle.Most of these kids are in diapers and have feeding tubes. There are so many extra layers to consider. With our previous provider, my inbox was constantly flooded with five-alarm fire situations—visa issues, incorrect flight bookings, missing reimbursements. Patients would reach out to the site first, but when they didn’t get a response, they’d come to me. I don’t get those emails anymore. That tells me everything.”
“Talk about an extraordinary turnaround.”
- Myrtelle’s Head of Community & Communications
She recalled attending a conference where trial participants shared their experiences.“Families told me, completely unprompted, that working with Mural Health has been incredible. Everything has been smooth—flights, travel, reimbursements. That was the first time I heard direct, glowing feedback.I saw it in their faces. That’s when I knew we had made the right decision partnering with you.”
She added that what’s most incredible is the commitment these families have to returning.
“No family is legally required to continue in the study–they can drop out at any time. But they don’t. They believe in science, and they understand that the data they’re providing could help the next child who receives this diagnosis. That commitment is inspiring.Supporting them with kindness and care helps make that possible.”
She added that what’s most incredible is the commitment these families have to returning.
“No family is legally required to continue in the study–they can drop out at any time. But they don’t. They believe in science, and they understand that the data they’re providing could help the next child who receives this diagnosis. That commitment is inspiring.Supporting them with kindness and care helps make that possible.”
“The immediacy with whichMural Health reimburses families makes a world of difference.”
- Myrtelle’s Head of Community & Communications
The Head of Clinical Operations also noted that the Mural Link portal is “the most user-friendly system” she’s ever seen.
“It took me two minutes to figure it out, and I’m not the most technical person in the world.Families upload their receipts, and everything is categorized clearly. It’s seamless. More importantly, families can get their reimbursements immediately. That’s not just convenient–it’s essential.”
Beyond seamless, immediate, and fee-free payments, Mural Health provides personalized support through its Patient Kindness Group, ensuring families navigate complex travel logistics, receive personalized assistance, and truly feel valued and seen.
“It took me two minutes to figure it out, and I’m not the most technical person in the world.Families upload their receipts, and everything is categorized clearly. It’s seamless. More importantly, families can get their reimbursements immediately. That’s not just convenient–it’s essential.”
Beyond seamless, immediate, and fee-free payments, Mural Health provides personalized support through its Patient Kindness Group, ensuring families navigate complex travel logistics, receive personalized assistance, and truly feel valued and seen.
“The biggest game-changer isthat now families have someonethey can talk to. It’s personal.
- Myrtelle’s Head of Community & Communications
“Every family feels seen. Every family feels heard. Every family receives individual attention, which was completely lacking before. We’ll hear stories about participants who shared with you that they had a hard day, and you sent them a little gift or a toy.It’s a beautiful model that Mural Health has created. It’s like that Mastercard commercial–what you do is priceless. These are incredibly sick kids we’re supporting. They need tender care, and you help us deliver on that.”
Myrtelle recalled one situation where a family from another country faced unexpected travel disruptions, and Mural Health stepped in.
“There was a child in a specialized chair, and their flight was canceled after waiting three hours on the runway. Instead of leaving them stranded or forcing them to figure it out alone,Mural stepped in. You helped them navigate rescheduling their flight, ensured they had a safe place to stay, and got them to their treatment. And your team did all of this after hours, completely outside of business timezones. That kind of responsiveness is rare.”
“There was a child in a specialized chair, and their flight was canceled after waiting three hours on the runway. Instead of leaving them stranded or forcing them to figure it out alone,Mural stepped in. You helped them navigate rescheduling their flight, ensured they had a safe place to stay, and got them to their treatment. And your team did all of this after hours, completely outside of business timezones. That kind of responsiveness is rare.”
“You have brought decency and respect to our patients and their caregivers.”
- Myrtelle’s Head of Community & Communications
She emphasized how these moments—often unseen by the broader clinical research industry—can determine whether a family stays engaged in a study. Because at the end of the day, trials depend on participation.If families feel unsupported, they are less likely to continue. And without their involvement, research is stunted or halted completely.
“With our prior vendor, that family might have turned around and gone home. But with MuralHealth, they stayed the course and got their child the care they needed.”
“You’re not just processing payments. You’re helping us ensure they make it to the trial.And that changes everything.”
“With our prior vendor, that family might have turned around and gone home. But with MuralHealth, they stayed the course and got their child the care they needed.”
“You’re not just processing payments. You’re helping us ensure they make it to the trial.And that changes everything.”
“I recently told someone in the industry that if you want your families to feel like they’re the only priority in that moment, Mural Health is the right partner.”
- Myrtelle’s Head of Community & Communications
“Every detail matters. Some of these families have specific dietary needs—like specialized formulas—and you guys go out of your way to accommodate them. I know of a case where one family wanted to bring back formula from the U.S. that they couldn’t access in their home country. Mural Health jumped right in and coordinated with the site to ensure it was safely shipped to their home. You care about our families having access to exactly what they need. Above and beyond seems to be your standard.”
For a clinical operations team, this kind of proactive support and reliability alleviates significant stress–not just for the participant experience, but for the entire trial process.
“As a Sponsor, when I see families being taken care of like that, it’s one less thing I have to worry about. It means I can focus on worrying about the science instead. I’m not chasing down patient travel issues because of logistical failures. I know Mural Health is taking care of it. The peace of mind you bring me? I haven’t had it for years. And it makes all the difference.”
For a clinical operations team, this kind of proactive support and reliability alleviates significant stress–not just for the participant experience, but for the entire trial process.
“As a Sponsor, when I see families being taken care of like that, it’s one less thing I have to worry about. It means I can focus on worrying about the science instead. I’m not chasing down patient travel issues because of logistical failures. I know Mural Health is taking care of it. The peace of mind you bring me? I haven’t had it for years. And it makes all the difference.”
“If patients aren’t at the center of everything we do, we won’t get the data we need.”
As both clinical trials and technology continue to evolve, it’s critical we not lose sight of our shared humanity. Compassion is just as crucial as innovation. By prioritizing the participant experience, Mural Health is redefining participant support and setting a new standard for what participant-centered clinical research should be.
“Ultimately, we can’t go in front of the FDA and say, ‘It was too hard for families to travel.’ If patients aren’t at the center of everything we do, we won’t get the data we need,” said the Head of ClinicalOperations. “The whole reason we’re doing this is to improve these families’ lives. If we can ease their burden in anyway while they’re being treated, it’s incredibly powerful. If families are more motivated because they’re seen, heard, respected, and treated with kindness and sensitivity, they show up. That’s how we get the data. Without reliable travel and reimbursement processes, these essential follow-ups were at risk. So as a Sponsor, we can’t put a price on the value you bring us.”
For Myrtelle’s Co-Founder and Head of Community & Communications, this trial is deeply personal:
“When my son was diagnosed with this same rare disease 30 years ago, I asked doctors about gene therapy. They said it was considered ‘pie in the sky’ and to not waste my time chasing false hope. But I didn’t accept that. I mobilized, found researchers working on gene therapy, and fought for change. I knew the science was in its infancy and likely wouldn’t be ready in my son’s lifetime, but it might ease his symptoms—and crack open the door for future generations.”
Today, the science she helped champion is directly targeting the brain cells these children need to restore function. What was once considered “a pie in the sky” is now precision medicine, and has helped pave the way for other gene therapies being studied for this disease today. That’s what research is all about—advancing science not just for today’s patients, but for the families who don’t yet know they’ll need it.
“Ultimately, we can’t go in front of the FDA and say, ‘It was too hard for families to travel.’ If patients aren’t at the center of everything we do, we won’t get the data we need,” said the Head of ClinicalOperations. “The whole reason we’re doing this is to improve these families’ lives. If we can ease their burden in anyway while they’re being treated, it’s incredibly powerful. If families are more motivated because they’re seen, heard, respected, and treated with kindness and sensitivity, they show up. That’s how we get the data. Without reliable travel and reimbursement processes, these essential follow-ups were at risk. So as a Sponsor, we can’t put a price on the value you bring us.”
For Myrtelle’s Co-Founder and Head of Community & Communications, this trial is deeply personal:
“When my son was diagnosed with this same rare disease 30 years ago, I asked doctors about gene therapy. They said it was considered ‘pie in the sky’ and to not waste my time chasing false hope. But I didn’t accept that. I mobilized, found researchers working on gene therapy, and fought for change. I knew the science was in its infancy and likely wouldn’t be ready in my son’s lifetime, but it might ease his symptoms—and crack open the door for future generations.”
Today, the science she helped champion is directly targeting the brain cells these children need to restore function. What was once considered “a pie in the sky” is now precision medicine, and has helped pave the way for other gene therapies being studied for this disease today. That’s what research is all about—advancing science not just for today’s patients, but for the families who don’t yet know they’ll need it.
