Enrolling by invitationPHASE1, PHASE2NCT05152823

Gene Therapy for IGHMBP2-Related Diseases

Studying Spinal muscular atrophy with respiratory distress type 1

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Key facts

Sponsor
Megan Waldrop
Principal Investigator
Megan Waldrop, MD
Nationwide Children's Hospital
Intervention
Gene Therapy(biological)
Enrollment
10 enrolled
Eligibility
14 years · All sexes
Timeline
20212030

Study locations (1)

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

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