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 (NASDAQ:SRPT) stock draws an Overweight rating at Wells Fargo, based on commercial prospects for ...

Explore Solid Biosciences’ potential with promising SGT-003 trial results for DMD, strong financials, and market recovery ...

A few weeks after Sarepta Therapeutics confirmed a patient died after receiving its drug Elevidys, research published April 3 ...

The findings suggest that the body's own immune system may be undermining the treatment with gene therapy such as Elevidys ...

The determination comes after recruitment and dosing in certain trials were paused after a young patient died due to acute ...

One day after the European Medicines Agency requested that three clinical trials of Elevidys be placed on hold after the ...

The firm said the clinical trial pause is temporary as an independent data monitoring committee finalizes its analysis for submission to regulators.

Therapeutics shared the following update related to ELEVIDYS, the only approved gene therapy in patients with Duchenne muscular ...