Gene Therapy May Improve Vision in XLRP, Further Research Needed
Serious adverse events of reduced visual acuity and eye inflammation were observed in 2 clinical trials.
RP-A501 Gene Therapy Treatment Well-Tolerated in Male Patients with Danon Disease
The low-dose cohort of RP-A501 conferred cardiac LAMP2B transgene expression.
In-Human Gene Therapy BAY 2599023 Effective in Patients with Severe Hemophilia
A new study presented at ASGCT found that patients with indications of hemostatic efficacy experienced few adverse effects with gene therapy treatment BAY 2599023.
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