Multidisciplinary coordination across prescribing teams, nursing, laboratory medicine, finance, and infusion centers is crucial for gene therapy delivery in Duchenne muscular dystrophy (DMD).

Just a few weeks after closing a $155 million series B, RNA editing biotech AIRNA is bringing an experienced gene therapy ...

The prospects for a one-time treatment that reduces the risk of heart disease for the rest of a person’s life just got ...

Getting therapeutic drugs past the blood-brain barrier has long been a major challenge in treating brain diseases. Now, researchers have explored how cholesterol-modified heteroduplex oligonucleotides ...

Sarepta Therapeutics SRPT announced updates from its clinical programs, which focus on targeting different subtypes of limb-girdle muscular dystrophy (LGMD), a group of rare genetic disorders marked ...

A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the way for better, more effective treatments.