Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy is on hold due to safety issues. An FDA official warns the path to market approval is fraught with challenges, especially after patient deaths linked to liver injuries.
In a stunning move, the FDA has put clinical trials for Sarepta's gene therapy on hold due to alarming safety concerns after multiple patient deaths linked to acute liver failure. The company faces scrutiny as it navigates the fallout.
Sarepta Therapeutics is under fire as another patient succumbs to acute liver failure linked to its gene therapy trial for muscular dystrophy, adding to the recent deaths of two teenage boys.
Franklin W. Stahl, renowned for his groundbreaking work on DNA replication, has died at 95. His 1957-58 experiment with Matthew Meselson reshaped the understanding of genetics and paved the way for future discoveries.
Researchers at the Peter Doherty Institute have made a groundbreaking discovery that makes the HIV virus visible, paving the way for potential cures. This innovative approach using lipid nanoparticles could revolutionize treatment.
In an unprecedented medical achievement, a child with a rare genetic disorder receives a customized CRISPR gene therapy, paving the way for future treatments of similar conditions.
Researchers have developed a groundbreaking method for inserting large genes into specific locations in human cells, potentially transforming the field of gene therapy and opening new avenues in medical research.
In a groundbreaking case, a 6-month-old baby boy named KJ was treated with a personalized CRISPR gene-editing therapy, correcting a dangerous mutation before it could threaten his life. This innovative approach promises hope for similar cases.
A baby diagnosed with a rare genetic disorder has shown remarkable improvement after receiving an experimental gene editing treatment tailored just for him. This groundbreaking therapy offers hope for others with similar conditions.
Emily Kramer-Golinkoff, a cystic fibrosis patient with a rare mutation, highlights the struggles of those left behind as gene therapies show promise for others. Her journey underscores the need for equitable treatments in rare genetic conditions.
