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 brighter. In an initial trial, a single dose of a CRISPR gene-editing therapy ...

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 ...

Oppenheimer analyst Andreas Argyrides lowered the firm’s price target on Sarepta (SRPT) to $184 from $215 and keeps an Outperform rating on the ...

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