Proven in rare neuromuscular and pediatric trials. For your Duchenne study, we reach families through the communities they trust and support them through every step.

Leapcure has recruited across rare neuromuscular and pediatric trials, from Myasthenia Gravis rescues to a Phase 3 study in children ages 2 to 11. For Duchenne, our dedicated Patient Success team supports each family with sensitivity, and we deliver enrollment-ready patients to your sites.

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130+
Studies recruited
across rare and complex disease
50+
Countries reached
for dispersed rare populations
1,600+
Clinical trial sites supported
across our programs
12,000+
Global advocacy partnerships
the network that reaches rare communities

Understanding Duchenne Muscular Dystrophy as a Clinical Trial Indication

Duchenne muscular dystrophy (DMD) is a rare, X-linked neuromuscular disease caused by mutations in the dystrophin gene, which leave muscle unable to produce functional dystrophin and lead to progressive muscle degeneration. It affects primarily boys, with an incidence of about 1 in 3,500 to 3,600 male births (StatPearls; PubMed). US diagnosed prevalence is roughly 6 per 100,000 males, about 10,000 people (Value in Health, 2019). Symptom onset is in early childhood, typically between ages 3 and 5, with most boys diagnosed around age 5. About 64.5 percent of patients are under age 20, and median life expectancy ranges from the teens to the late 30s depending on ventilatory support (Value in Health, 2019; systematic review, 2023). Caregiver burden is higher than for many other neuromuscular disorders.

For sponsors, Duchenne is one of the harder rare indications to enroll. The eligible population is small and dispersed, and because patients are children, every enrollment decision is made by parents and caregivers who are weighing a serious, progressive diagnosis. Trust matters more than reach. Eligibility is often narrowed further by age band, ambulatory status, prior or current therapy, and specific mutation types, so the patients a given protocol needs are a small slice of an already rare population. Families also cluster around a limited number of neuromuscular centers and the advocacy organizations they already rely on, which is where eligible, motivated families can actually be found.

Leapcure recruits for Duchenne the way these families decide: through the communities and advocacy networks they already trust, with a dedicated Patient Success team built for the sensitivity a childhood disease requires. Our experience spans rare neuromuscular trials, including Myasthenia Gravis, ALS, Charcot-Marie-Tooth, and dermatomyositis, and pediatric, family-decided rare disease, including a completed Phase 3 study in children ages 2 to 11. Across our programs we have recruited for 130+ studies, supported 1,600+ sites in 50+ countries, and built 12,000+ global advocacy partnerships. We pre-screen and validate every referral before it reaches a site, so coordinators receive enrollment-ready families, not referral volume.

The Challenge

Duchenne recruitment is a trust problem before it is a reach problem. The eligible population is small and dispersed, the patients are children, and the people who decide are parents weighing a serious diagnosis.

Duchenne is a family decision before it is a patient decision

Duchenne patients are children, so enrollment is decided by parents and caregivers, often while they are still absorbing a progressive diagnosis. Generic outreach that treats recruitment as a transaction does not earn that trust. Families need honest information, time, and a single person who supports them through the decision, whichever way it goes.

On a Phase 3 study in children ages 2 to 11, our team held 30+ in-depth conversations with parents and built lasting trust even with families who chose not to proceed.

The eligible population is tiny and globally dispersed

US diagnosed prevalence is roughly 6 per 100,000 males, about 10,000 people, and protocols narrow that further by age, ambulatory status, prior therapy, and mutation type. Finding enough eligible boys for a Duchenne trial means reaching far beyond any single region or site catchment.

Leapcure reaches dispersed rare populations across 50+ countries through 12,000+ global advocacy partnerships.

Families are reached through communities, not list buys

Duchenne families gather in disease-specific advocacy organizations and caregiver communities they already trust. Patients are rarely found through cold digital or purchased lists. Recruitment works when research reaches families through the voices they already rely on.

The Muscular Dystrophy Association (MDA) is our named muscular dystrophy advocacy partner, and we reach the wider Duchenne community through our broader rare-disease network.

Referral volume is not the same as enrollment-ready patients

A flood of unvalidated referrals buries site coordinators and inflates screen fails. Duchenne sites are specialized and busy, and their time is the constraint. The patients who arrive need to be pre-screened, eligibility-reviewed, and prepared for what a trial involves before a coordinator ever picks up the phone.

Triple validation, adaptable pre-screening, human one-to-one conversations, and an in-house MedOps clinical-eligibility review, happens before any site contact.

How Leapcure recruits for your Duchenne study

We reach Duchenne families through the communities they trust, validate every referral before it reaches a site, and support each family from first contact through randomization.

1

Activate the communities Duchenne families trust

We engage the Muscular Dystrophy Association and our broader rare-disease and rare-neuromuscular advocacy network, alongside precision digital and physician outreach mapped to your eligibility criteria, age bands, sites, and geographies. Research reaches families through sources they already rely on, not unsolicited pitches.

2

Validate every referral before it reaches a site

Each family goes through triple validation: adaptable pre-screening, real one-to-one conversations rather than automated screening, and an in-house MedOps clinical-eligibility review. Sites conduct official screening. We make sure the families who arrive are informed, eligible, and prepared.

3

Support each family through randomization

A dedicated Patient Success Coordinator is each family's single point of contact, with documentation-ready site handoff and continuous support through randomization. This is built for the sensitivity Duchenne requires: families are the decision-makers, the patient is a child, and trust is everything.

Our closest analog to Duchenne: a Phase 3 study in children ages 2 to 11, where family trust drove every referral.

Challenge Context

Indication

Pediatric, family-decided rare disease (Phase 3, children ages 2 to 11)

Sponsor

Leading rare-disease sponsor

Challenge

A tiny pediatric population, dispersed across regions, where every decision sat with parents and caregivers weighing a serious diagnosis for a young child.

Channels Deployed

Disease-specific advocacy groups, physician engagement across states, precision digital, and a family-sensitive Patient Success team with documentation-ready site handoff.

Results & Metrics

Family Engagement

200+ referral attempts and 30+ in-depth conversations with parents, with a compassionate approach that built long-term trust even with families who did not proceed.

Physician and Advocacy Reach

70+ physicians engaged across 18 states and 20+ advocacy groups engaged, with families committed to site screening.

Rare-neuromuscular track record

On a Myasthenia Gravis Phase 2 rescue, Leapcure delivered 8 of the final 10 participants in the closing months; on a Phase 3 dermatomyositis study, 4 of 9 sites enrolled only Leapcure-referred patients.

Why it transfers to Duchenne

Duchenne is decided by families, for a child, under real weight. The pediatric Phase 3 analog and our rare-neuromuscular results show the family sensitivity and the validation rigor a competitor cannot fake.

Four channels. Built for Duchenne families. Active before your trial opens.

Leapcure reaches Duchenne families through trusted advocacy communities, a family-first Patient Success team, precision digital and physician engagement, and answer-engine visibility delivered to your trial.

Channel 1: Advocacy and community network

  • •The Muscular Dystrophy Association (MDA) is our named muscular dystrophy advocacy partner, serving the Duchenne community directly.
  • •We reach the wider Duchenne community through our broader rare-disease network, with 12,000+ global advocacy partnerships across 50+ countries.
  • •Content is co-developed with advocacy partners so research reaches families through sources they already trust.

Channel 2: Family-first Patient Success team

  • •A dedicated Patient Success Coordinator is each family's single point of contact, from first conversation through randomization.
  • •Triple validation, adaptable pre-screening, human conversations, and an in-house MedOps clinical-eligibility review, happens before any site contact.
  • •This is the human differentiator for Duchenne: dedicated support built for the sensitivity a childhood disease requires.

Channel 3: Precision digital and physician engagement

  • •Targeted digital outreach mapped to eligibility, age bands, and site proximity, reaching families where they already are.
  • •Direct physician engagement surfaces diagnosed Duchenne patients through their treating neuromuscular clinicians.
  • •Channel mix is rebalanced by referral quality and site capacity so referrals stay matched to where sites can screen.

Channel 4: Answer-engine and AI-search visibility

  • •Families increasingly ask ChatGPT, Perplexity, and Google's AI Overview which trials a child may be eligible for. We make your trial's information genuinely retrievable for those engines.
  • •We do this with crawlable, factually rich content, cited sources, and presence in the advocacy and community sources AI engines actually cite, not schema tricks or content written for machines.
  • •Because AI engines cite trusted third-party sources more often than any single brand domain, our advocacy partnerships and ClinicalTrials.gov presence are the structural advantage we bring to your trial.

Advocacy depth across rare neuromuscular disease

The Muscular Dystrophy Association is our named muscular dystrophy partner, serving the Duchenne community directly, and we reach the wider Duchenne community through our broader rare-disease network of 12,000+ global advocacy partnerships. Our rare-neuromuscular advocacy depth shows what that network delivers.

Muscular Dystrophy Association (MDA)

Our named muscular dystrophy advocacy partner, a leading organization that serves the Duchenne community directly

Myasthenia Gravis advocacy network

30+ MG advocacy partnerships, mobilizable in two days, that supported a Phase 2 rescue

ALS advocacy network

About 100 advocacy partnerships across 10+ countries that supported a global Phase 3 program

Dermatomyositis advocacy network

30+ engaged myositis advocacy partners that drove the majority of enrollment on a Phase 3 study

Global rare-disease network

12,000+ global advocacy partnerships across 50+ countries reaching small, dispersed rare populations

Caring for a child with Duchenne? A clinical trial may be an option to explore.

If you are a parent or caregiver of a child with Duchenne muscular dystrophy, a clinical trial may offer access to investigational treatments alongside your child's existing neuromuscular care, never instead of it. We know this is a heavy decision, and we will never pressure you. Leapcure's team answers your questions honestly, explains what a trial involves, and supports your family at your pace, whatever you decide.

Learn about Duchenne trials for your family
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Leapcure recruits for Duchenne on proven rare-neuromuscular and pediatric experience, from a Myasthenia Gravis rescue to a Phase 3 study in children ages 2 to 11, with the Muscular Dystrophy Association as our named partner and a family-first Patient Success team built for the sensitivity Duchenne requires. We reach families through the communities they trust, validate every referral before site contact, and deliver enrollment-ready patients to your sites. We can mobilize quickly.

Talk to our team