Ask Max: Clinical Trials – Our Family’s Journey

Your monthly Q&A with Maxine—Duchenne parent and HF Fundraising & Chrysalis Grant Manager

Last month we launched Ask Max—a space where the Duchenne community can send in real questions and hear directly from someone who’s living it. This month, Maxine responds to a recurring theme: Should we consider a clinical trial? What’s it really like? Below, she shares her family’s honest experience after more than eight years in a trial with her son, Austin.

Maxine writes:

Clinical Trials: Our Family's Journey — The Highs, the Lows, and Why We Keep Going

When our son Austin was first diagnosed, we found ourselves in a world with more questions than answers. Options felt limited. That’s when a clinical trial became a beacon of hope.

We've now been part of a clinical trial for over eight years. It’s been a journey marked by early mornings, long drives, tough procedures, but also incredible support, progress, and purpose. We want to share both the rewards and the realities, so other families considering this path know they’re not alone.

The Pros: Why We’re Grateful for the Trial

1. Access to cutting-edge treatments

The trial gave us early access to a therapy still under investigation, something we otherwise wouldn’t have had. When treatment options are few, that kind of opportunity means everything.

2. Exceptional medical oversight

The level of care in a clinical trial is second to none. Austin’s health has been monitored incredibly closely. In fact, trials often detect underlying health issues early, thanks to the frequency and depth of assessments.

3. Feeling part of something bigger

It’s hard to describe the sense of purpose that comes with contributing to something that may help others in the future. Every blood test, every visit—it’s all part of moving Duchenne care forward.

4. Building lasting relationships

Over time, we’ve developed deep trust and friendships with the research team. The staff at the Clinical Research Facility in Leeds have been phenomenal, supportive, compassionate, and truly invested in Austin’s wellbeing.

5. Resilience and routine

Surprisingly, Austin enjoys the process. Those 4 a.m. starts? He meets them with enthusiasm. It’s become a meaningful part of his life—a sense of control and adventure in something otherwise so uncertain.

The Cons: What Makes It Hard

1. The burden on family life

Weekly dosing meant a complete upheaval of our routine, impacting school attendance, work, and family time. The travel alone (197 miles each way) made for 14-hour days, sometimes every week.

2. Invasive procedures

Clinical trials involve regular tests, blood tests, heart scans—even biopsies. These aren’t easy, especially for a child—and yet, Austin has met each one with courage.

3. Emotional ups and downs

There’s hope, but also heartbreak. It’s painful to know the treatment might not be a cure, or that others may not yet have access. There’s joy in progress, but a constant, quiet grief in how slow it feels.

4. Uncertainty

Trials come with unknowns—outcomes, side effects, timelines. Living in a state of “wait and see” can be emotionally exhausting.

What Keeps Us Going

Through it all, Austin’s strength and positive spirit guide us. We’ve become part of a community—one that laughs together in hospital corridors, swaps travel tips, and shares updates like family. We feel the weight of the work, but also the immense value of it.

Each trial brings us one step closer to a breakthrough. Today’s data shapes tomorrow’s treatments and while we wish things were moving faster, there’s real hope in the Duchenne community, and we’re proud to be part of that momentum.

We share our story so others might feel less alone in theirs. Clinical trials are demanding, but deeply meaningful. And for us, they’ve brought not just medical progress, but connection, courage, and an unexpected kind of joy.

Austin’s already looking forward to his next “Leeds adventure.” 😊

Thinking About a Clinical Trial?

Questions to Ask

If your clinical team raises a trial option—or you’re researching one—these conversation starters (inspired by Max’s experience) may help:

• What stage is the trial in, and what’s known about safety so far?

• Is there a placebo arm? crossover?

• How often are visits, and where? (Travel matters!)

• What procedures are required (bloods, biopsies, imaging)?

• How will trial participation affect school attendance and daily life?

Can data from the trial benefit my child’s care, even if the treatment doesn’t?

Have a question for Max about clinical trials—or anything else related to life with Duchenne? Send it to us and it may feature in a future Ask Max. You’re never on this road alone.

With care,

Maxine