Scientists took skin cells from two people whose hearts were growing too thick, which can cause dangerous rhythms and block blood flow. Inside each cell’s instruction manual, a single wrong letter in the PRKAG2 gene was spotted—like a typo in a recipe that makes the heart muscle store extra sugar in
A study followed people with a specific genetic hearing loss called OTOF‑related deafness for two and a half years after they received a gene therapy. The treatment used a harmless virus to deliver a healthy copy of the missing gene into inner‑ear cells. Researchers checked many things over time, su
Every year, science gets its own red-carpet moment in Santa Monica. A mix of Hollywood heavyweights, tech leaders, and top researchers gathered to celebrate the most exciting work in math, physics, and life sciences. The Breakthrough Prize has become a unique event where brainpower meets glamour. Wh
Duchenne muscular dystrophy is a tough disease that makes muscles weak over time. Kids with this condition often struggle with simple movements like standing up or walking. But there's some good news! A treatment called ELEVIDYS is showing positive results in a big study called EMBARK. The study lo
Genenta Science, a company working on new ways to fight cancer, has some news to share. Their CEO, Pierluigi Paracchi, has been buying up shares of the company. Over time, he's picked up 30, 000 American Depositary Shares, or ADSs, through open-market purchases. No sales have been reported, just buy
Two biotech startups, RenBio and Fractyl Health, are exploring innovative ways to help the body produce more GLP-1, a hormone that plays a crucial role in regulating appetite and blood sugar levels. Their goal is to develop gene therapies that can turn cells into tiny factories producing GLP-1, pote
Vinay Prasad is back at the Food and Drug Administration (FDA), picking up where he left off just two weeks ago. He's in charge of vaccines, gene therapies, and blood products again. The FDA asked him to come back, but it's not clear if he'll keep his other big job as the agency's top medical and sc
Mitochondria are like tiny powerhouses inside our cells. They keep us going, but sometimes they go wrong and cause problems. Tiny molecules called miRNAs help control these powerhouses. Scientists have been trying to find ways to see and fix these miRNAs to help treat diseases like cancer. A new id
In a surprising turn of events, Sarepta Therapeutics has chosen to defy the FDA's request to stop distributing its gene therapy drug, Elevidys. This decision comes after three patient deaths linked to the treatment. The FDA has expressed serious concerns about the safety of the drug and has asked Sa
In Italy, a big question is being asked about a new treatment for babies born with a rare condition called spinal muscular atrophy (SMA). This condition affects the nerves that control muscles, making it hard for babies to move, breathe, and even swallow. The treatment in question is called onasemno
