For pharma & research
Reach the right patients, faster.
Improve trial visibility, accelerate recruitment, and access real-time engagement data across the Duchenne community.
Trial Visibility
Surface your trial to qualified Duchenne families across geographies — not just at top-tier sites.
Smarter Recruitment
Reduce screen-fail rates with patient profiles enriched by clinical, genetic, and ambulatory data.
Real-Time Insights
Anonymized engagement, sentiment, and journey data — directly from the community.
Why DMD Pathways
Built for the realities of rare disease research.
Duchenne is heterogeneous, geographically dispersed, and time-sensitive. Traditional recruitment channels weren't built for it. We were.
70%+
Of DMD families want trial info but don't know where to find it
<2%
Of eligible patients are typically reached for rare disease trials
1:1
Direct, opted-in family engagement
Global
Trial discovery across 40+ countries
Patient Engagement
Build trust with Duchenne families — long before enrollment opens.
In rare disease, the families who enroll are the families who already know you, trust your science, and feel respected by your team. DMD Pathways is the connective tissue between sponsors and the Duchenne community — a continuous, consented engagement layer that runs alongside your program from preclinical through post-approval.
Two-way family dialogue
Plain-language program updates, Q&A sessions, and structured feedback loops with caregivers and adult patients.
Trial education that lands
Mutation-aware explainers, visit-burden previews, and consent walkthroughs co-designed with DMD families.
Caregiver-first support
Logistics, travel, and emotional support resources so participating in research doesn't break the family.
Community co-creation
Patient advisory input on protocol design, endpoint selection, and recruitment messaging before you go live.
Global reach, local nuance
Engagement across 40+ countries with culturally and linguistically adapted touchpoints.
Site-aware handoffs
Warm referrals to your activated sites with the clinical context they need to pre-screen efficiently.
The engagement flywheel
Listen, educate, activate — then learn and repeat.
Every interaction with a family generates de-identified signal: what they understand, what worries them, what barriers stand between them and your trial. We turn that signal into protocol-ready insight your team can act on.
Listen
Structured caregiver intake & sentiment
Educate
Plain-language trial & disease content
Activate
Consented matches to your sites
Learn
Insights back to your medical & clinical teams
Retention & Human Navigation
A human layer that keeps families in your trial — and your trial on track.
Recruitment is only half the battle. The real cost of rare disease trials is lost at the margins: families who screen-fail because of a misunderstanding, drop out after the first visit, or deviate from protocol because no one checked in. DMD Pathways adds a dedicated human navigation layer between sponsor, CRO, and site teams and the families you serve — so enrollment converts to completion.
Human navigation layer
Dedicated care navigators who walk families through eligibility, consent, scheduling, and ongoing trial logistics — not chatbots, real people.
Faster time-to-enroll
Pre-qualified, educated families arrive at sites ready to screen. Navigators handle paperwork, travel, and questions before the first visit.
Retain enrolled patients
Proactive check-ins, caregiver support, and barrier removal so families stay engaged through long, demanding protocols — not just the easy visits.
Fewer screen failures
Navigators verify genetic eligibility, mutation fit, and inclusion criteria with families before they ever reach your site.
Reduce dropouts & deviations
Early warning signals from family touchpoints flag travel strain, comprehension gaps, or care-team friction before they become protocol deviations.
Site-ready handoffs
Warm referrals with full family context, clinical history, and documented barriers so sites spend less time chasing and more time treating.
Value for every stakeholder
Sponsors, CROs, and sites — each get a partner, not another platform.
For Sponsors
- Hit enrollment targets on time
- Lower cost-per-randomized patient
- De-risk protocol with family insight
- Protect data integrity through retention
For CROs
- Pre-qualified referral pipeline
- Fewer screen-fail surprises
- Real-time enrollment & retention dashboards
- Dedicated navigator as site extension
For Sites
- Warm families who understand the trial
- Logistics handled before arrival
- Ongoing caregiver support between visits
- Early alerts on at-risk participants
Sponsor Intelligence Portal
See the operational story from
first inquiry through study completion.
The DMD Pathways Sponsor Intelligence Portal is being designed to give sponsors, CROs, and research teams a clearer view of the family experience across recruitment, enrollment, participation, and retention.
Behind the portal is a human layer of navigation. DMD Pathways navigators maintain ongoing contact with participating families, help them work through practical barriers, and reduce the communication and coordination burden placed on site staff.
With appropriate consent and data governance, those family interactions can also surface emerging risks before they become missed visits, protocol deviations, disengagement, or withdrawal—so study teams can respond earlier and keep families supported and engaged.
What sponsors can explore:
Inquiry-to-EnrollmentProgress
Understand how families move from initial interest through referral, screening, consent, and enrollment.
Ongoing PatientEngagement
Maintain a consistent human connection between site visits so families have a trusted place to raise questions and concerns.
Early Retention-RiskSignals
Identify changes in circumstances, confidence, burden, or engagement that may indicate a future missed visit, deviation, or withdrawal.
Navigator-SupportedInterventions
Track non-medical navigation actions that help families address barriers and determine when escalation to the site is needed.
Retention andParticipationTrends
View de-identified patterns in missed visits, deviations, disengagement, withdrawals, and recurring barriers.
Human navigation that supports families and sites
We support the clinical relationship. Navigators help families stay informed, prepared, and connected—reducing avoidable administrative and communication demands on site teams.
Navigators do not provide medical advice or make clinical decisions. Medical, safety, eligibility, or protocol concerns are directed back to the study team.
Data governance comes first
The portal is built around appropriate consent, permissioned access, data minimization, and de-identification.
Identifiable information is shared with the relevant study team only when authorized by the family and necessary for an agreed referral, escalation, or support process.
Any production implementation will be subject to privacy, security, data protection, medical, legal, and regulatory review.
Site-ready communication and trial-specific reporting
Provide sites with relevant, consented information so staff can focus attention where it's most needed.
Incorporate study-defined engagement measures, retention indicators, referral requirements, and sponsor reporting priorities.
Let's accelerate your next Duchenne program.
Schedule a confidential briefing with our partnerships team.
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