Key Takeaway
This is the main currently listed FKRP-targeted gene-therapy trial record and it is important because it shows who was eligible, what safety questions were being studied, and where the field stands clinically.
Trial and Study Record
ATA-100 (Formerly GNT0006) Gene Therapy Trial in Patients With LGMDR9
Phase 1 FKRP gene-transfer trial record for ATA-100, designed to assess safety, pharmacodynamics, and immunogenicity with long-term follow-up.
TrialTherapy DevelopmentTrial ReadinessFamilies
Why this record matters
- Directly relevant to the current therapeutic pipeline because it evaluates an FKRP-specific gene-transfer strategy rather than nonspecific supportive care.
- The record clarifies that this is an early safety-oriented trial with only six enrolled participants, so it should not be interpreted as proof of efficacy.
- Long-term follow-up through 2029 means the page is useful for orientation but still depends on later publications and registry updates.
Eligibility and participation notes
- Enrolled ambulant patients aged 16 years and older with genetically confirmed LGMDR9.
- Required moderate diaphragmatic muscle impairment.
- Excluded patients with detectable serum neutralizing antibodies against AAV9 or cardiomyopathy.
Limits and cautions
- Phase 1 design and very small enrollment limit what can be inferred about benefit.
- Exclusion of cardiomyopathy means the study population does not represent every person living with FKRP-related disease.
- Trial status and posted dates can change, so the source record remains the authoritative reference.
Primary Sources
Direct links for verification
Primary source used for the structured study summary on this page.
Related Content
Related routes inside the site
Study Record
Study of BBP-418 in Patients With LGMD2I
Phase 2 open-label ribitol study in FKRP-related LGMD2I/LGMDR9 that provides the main early clinical development context for the BBP-418 program.
Study Record
BBP-418 (Ribitol) FORTIFY Trial in LGMD2I/LGMDR9
Phase 3 randomized ribitol trial in FKRP-related LGMD2I/LGMDR9 and the most advanced current interventional program identified in this FKRP research sweep.
Study Record
A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
Phase 3 corticosteroid study record evaluating deflazacort in adults with FKRP-related LGMD2I, ultimately terminated early.