Neuroscience

A multicenter look at gene therapy for spinal muscular atrophy

In May 2019, the U.S. Food and Drug Administration (FDA) approved a gene replacement therapy for the inherited, progressive neuromuscular disease 5q-linked spinal muscular atrophy (SMA). Approval included all children with ...

Medical research

First evidence of clinical stabilization in Tay-Sachs

Preliminary data from an expanded access study of an investigational gene therapy in two patients with infantile Tay-Sachs disease indicates the potential to modify the rate of disease progression, according to an upcoming ...

Genetics

Promising gene replacement therapy moves forward

Research led by Dr. Krystof Bankiewicz, who recently joined The Ohio State University College of Medicine, shows that gene replacement therapy for Niemann-Pick type A disease is safe for use in nonhuman primates and has therapeutic ...

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