Intra-Articular FX201 Gene Therapy Shows Promise for Osteoarthritis
The therapy was generally well-tolerated and offered substantial pain relief in patients.
ARU-1801 Improves Clinical Outcomes in Sickle Cell Disease
Early results from the MOMENTUM study show that treated patients experienced fewer vaso-occlusive episodes and related hospital admissions.
ADVM-022 Gene Therapy for nAMD May Reduce Anti-VEGF Injection Burden
The single-injection investigative gene therapy may help with providing continuous expression of aflibercept.
Gene Therapy BIIB112 Promising for X-Linked Retinitis Pigmentosa
The AAV8 vector–based gene therapy was associated with improvements in 2 measures of visual function.
Understanding Fabry Disease: Perspectives from a Nephrologist
As a multi-systemic disorder, Fabry disease can impact the functions of various organs. As a genetic disorder, Fabry can impact the offsprings, parents, and relatives of the patient. Therefore, its effects are far-reaching.
Gene Therapy GS010 Safe, Well-Tolerated for LHON
No approved treatment for leber hereditary optic neuropathy is currently available in the United States.
LentiGlobin Gene Therapy for Sickle Cell Demonstrates Therapeutic Potential
Data from the ongoing HGB-206 study reports reduced hemolysis, increased total hemoglobin, and a complete resolution of vaso-occlusive crises among patients.
Single-Cell RNA Sequencing May Improve Personalized Uveitis Therapy
Lynn Hassman, MD, talks about a promising new technology that can optimize precision medicine in patients with uveitis.
New Data Shows Promise for Investigative XLRP Gene Therapy
The study presented at AAO provides an update on previously shared 6-month data from earlier this year.
Risk Based Therapy Approach for Sickle Cell Anemia Patients is Ideal Scenario
The complexities associated with the disease and lack of validated risk predictors makes such a model challenging to implement.
FDA Issues CRL for Hemophilia A Gene Therapy
Valoctocogene roxaparvovec would be the first ever approved gene therapy to treat patients with hemophilia A.
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