Jonathan Alicea

The therapy was generally well-tolerated and offered substantial pain relief in patients.

Early results from the MOMENTUM study show that treated patients experienced fewer vaso-occlusive episodes and related hospital admissions.

The single-injection investigative gene therapy may help with providing continuous expression of aflibercept.

The AAV8 vector–based gene therapy was associated with improvements in 2 measures of visual function.

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.

No approved treatment for leber hereditary optic neuropathy is currently available in the United States.

Data from the ongoing HGB-206 study reports reduced hemolysis, increased total hemoglobin, and a complete resolution of vaso-occlusive crises among patients.

Lynn Hassman, MD, talks about a promising new technology that can optimize precision medicine in patients with uveitis.

The study presented at AAO provides an update on previously shared 6-month data from earlier this year.

The complexities associated with the disease and lack of validated risk predictors makes such a model challenging to implement.

Valoctocogene roxaparvovec would be the first ever approved gene therapy to treat patients with hemophilia A.