Jessica Kim Cohen

Drugmakers and researchers shared updates from research programs on rare diseases like Duchenne muscular dystrophy, Wilson's disease, and lysosomal disorders. 

The treatment has been used in a trial to treat children with GM1 gangliosidosis, but if the FDA allows, this would be the first time a gene therapy is given to a fetus.

Investigators presented interim data from a Phase III trial of Lenmeldy in patients with the late juvenile form of disease at the ASGCT conference.

From decoy DNA sequences to plasmapheresis, researchers are testing strategies to get around anti-AAV antibodies that hinder gene therapy response.

Most adults in the US would take a blood test to assess whether they will develop Alzheimer's, according to the survey, though experts urge caution.

The New York-based public benefit corporation is advancing three drug candidates for the extremely rare condition.

ACMG, NSGC, and others in the genetics community are monitoring whether the widely used online clinical genetics database could be at risk.

On the heels of its acquisition by Ocuphire Pharma, Opus is planning next steps for its Leber congenital amaurosis type 5 gene therapy.

The biotech firm closed an oversubscribed Series C funding round, proceeds from which it will use to advance ATSN-201 and its preclinical pipeline.

Researchers urged hospitals to set up processes for updating pharmacogenomic results, but it's unclear how frequently that should be done.