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Spinal Muscular Atrophy


Case Studies

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Key Takeaways

The study was evaluating an investigational therapy for spinal muscular atrophy (SMA) in newborns. To be eligible to participate, newborns had to be genetically diagnosed with SMA, pre-symptomatic and ≤ 6 weeks of age. 

Because of the rare nature of the disease and the narrow eligibility criteria, it was important that all potentially eligible patients were screened, and that those enrolled remained enrolled for the entire length of the study.

The study was considered a landmark study for SMA, and the investigational therapy has since been approved as the first treatment for SMA in pediatric patients.



Because newborns had to be ≤ 6 weeks of age at the time they received the first dose of the investigational therapy, early identification and action was essential.

Logistical and financial barriers had to be eliminated to ensure all potentially eligible patients could be considered for participation and, if enrolled, could participate for the length of the study.

Complicated international travel, often exceeding 500 miles one-way, had to be coordinated at an accelerated pace.

Solution and Impact

Logistical and financial barriers to participation were eliminated with BBK’s TCNTM Arrive travel program and TCNTM Card reimbursement program.

Travel support was customized based on individual needs and included visa procurement, patient ambassadors to assist with translation, international flights and long-term housing accommodations, as needed.

BBK’s concierge specialists ensured that no screening opportunity was missed, often coordinating complex travel support for visits occurring within 24-48 hours.

Reimbursements were immediate, contributing to overall study satisfaction.

comprehensive travel and reimbursement
  • Participants_ICON 6 Randomization needed to occur within six weeks of birth.

  • Increase-Referrals_ICON-1 19 At the time of BBK’s engagement, the client had enrolled six patients — BBK enrolled the remaining 19 patients needed to meet the enrollment goal of 25.

  • Calendar_ICON 8 The study enrolled eight months ahead of schedule.

TCNTM Arrive & TCNTM Card

  • 193 Itineraries Created

  • 79% International Travel

  • 1,227 Average Miles per Round-trip

  • 772 Visits Reimbursed

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