Spinal Muscular Atrophy Clinical Trial Patient Recruitment

SMA patient recruitment for Type 1, 2, 3, and 4 studies including gene therapy and antisense oligonucleotide trials.

Who qualifies: Patients with SMA (SMN1 deletion/mutation confirmed) across all types, including treatment-naive and treated patients.

Recruitment challenges: SMN1/SMN2 copy number requirements and prior nusinersen/risdiplam eligibility criteria.

Our approach: Neuromuscular disease center networks, SMA patient advocacy partnerships, and genetic pre-screening.