Clinical trials: LGMDR9
LGMDR9
ATA-100 (Formerly GNT0006) Gene Therapy Trial in Patients With LGMDR9
Phase 1 dose escalation study to assess tolerability and safety of ATA-100 with 5-year follow-up
- Trial name
- NCT05224505
- Testing
- ATA-100 (AAV9 encoding FKRP gene)
- Where
- 3 sites in Denmark, France and 1 more
- Volunteers
- 6 people
- Sponsor
- Atamyo Therapeutics
LGMD2I, LGMDR9
Global FKRP Registry
Mutations in the Fukutin Related Protein (FKRP) gene cause the condition Limb Girdle Muscular Dystrophy type R9 (LGMDR9) also known as LGMD2I, and the rarer conditions Congenital Muscular Dystrophy (MDC1C), Muscle Eye Brain…
- Conditions
- LGMD2I, LGMDR9, Limb Girdle Muscular Dystrophy, Congenital Muscular Dystrophy
- Trial name
- NCT04001595
- Testing
- Patient Registry
- Where
- United Kingdom
- Volunteers
- 1,000 people
- Sponsor
- Newcastle University