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 ...

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

Wall Street analysts said the results for Verve’s therapy appeared competitive on efficacy, while avoiding any major safety ...

The law firm of Kessler Topaz Meltzer & Check, LLP ( is currently investigating potential violations of the federal ...

Learn more about whether Alvotech or Sarepta Therapeutics, Inc. is a better investment based on AAII's A+ Investor grades, which compare both companies' key financial metrics.

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 ...