Beacon claimed the findings represent the first time a treatment for X-linked retinitis pigmentosa, a rare and potentially blinding condition, met its main goal in a pivotal trial.

FDA approves Fayuvi (rebisufligene etisparvovec-hopf), the first gene therapy for pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA).

A patient has died in a clinical trial evaluating a Rocket Pharmaceuticals gene therapy shortly after complications emerged that led the FDA to pause the study. An inquiry into the fatality is ...

Treatment is the first for MPS type IIIA ...

Stanford Health Care delivers the highest levels of care and compassion. SHC treats cancer, heart disease, brain disorders, ...

Before the end of September, UniQure is expected to report four-year results from the closely tracked clinical trial of its gene therapy for Huntington’s disease.

Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the long-term safety and efficacy of the curative treatment, in a study led by ...

Intramuscular injection of a gene therapy known as AMG0001 (Collategene, AnGes Inc.) significantly decreased healing time and increased healing rates of neuroischemic ulcers compared with placebo in ...

A study led by UMass Chan researchers demonstrated that a gene therapy to correct a mutation that causes maple syrup urine disease (MSUD) prevented newborn death, normalized growth, restored ...

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Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...

This article summarizes the current state of genetic testing and gene therapy based on the lecture by Shelby Redfield of ...