A team of researchers in Turkey have discovered multiple signs of stress in the eye after patients took Pfizer's COVID-19 ...

A team of researchers in Turkey have discovered multiple signs of stress in the eye after patients took Pfizer's COVID-19 ...

Duchenne muscular dystrophy families are sharing stories about Elevidys complications on social media. But they aren’t getting answers from Sarepta.

Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in connection with its gene therapy for muscular dystrophy.

Shares of Sarepta Therapeutics have plunged again after the drugmaker reported a second death in connection with its gene therapy for muscular dystrophy.

Biotech pauses trial after second patient death linked to gene therapy Sarepta Therapeutics said it would temporarily stop shipping treatments for certain Duchenne muscular dystrophy patients.

TGFBI corneal dystrophy is a group of genetic eye disorders caused by mutations in the TGFBI gene, resulting in abnormal protein buildup in the stromal layer of cornea. Symptoms include ...

About TGFBI Corneal Dystrophy TGFBI corneal dystrophy is a group of genetic eye disorders caused by mutations in the TGFBI gene, resulting in abnormal protein buildup in the stromal layer of cornea.

This trial marks the world's first clinical study of an in vivo CRISPR-Cas ribonucleoprotein (RNP)-based genome editing investigational therapy for TGFBI corneal dystrophy.

The research aimed to identify how neurodevelopmental and psychiatric conditions associated with dystrophinopathy, including DMD and Becker muscular dystrophy (BMD), were assessed.