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DRUG CLASS:
Frataxin gene expression enhancer
DRUG CLASS:
Frataxin gene expression enhancer
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Related drugs:
‹
GCSF (2)
GCSF (2)
›
Associations
(2)
News
Trials
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Latest
1year
SUNRISE-FA: Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia (clinicaltrials.gov)
P1/2, N=8, Active, not recruiting, Lexeo Therapeutics | Recruiting --> Active, not recruiting
1 year ago
Enrollment closed • Gene therapy
1year
Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia (clinicaltrials.gov)
P1, N=25, Recruiting, Weill Medical College of Cornell University | N=10 --> 25
1 year ago
Enrollment change • Gene therapy
|
AFP (Alpha-fetoprotein)
|
prednisone
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