LND topic: gene-therapy page 1

gene therapy

Genenta's CEO Invests Big: What It Means for the Company

December 20, 2025

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

health

Body's Own GLP-1: A New Hope for Weight Loss and Diabetes?

November 4, 2025

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

health

The Comeback Kid: Vinay Prasad's Unexpected FDA Return

August 9, 2025

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

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Smart Bubbles for Fighting Cancer and Seeing Inside Cells

July 29, 2025

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

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A Drug Company and the FDA in a Heated Dispute Over Patient Safety

July 20, 2025

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

health

New Hope for Babies: Weighing Costs and Benefits of a Rare Disease Treatment

June 26, 2025

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

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A Second Death Shakes Duchenne Community

June 17, 2025

The Duchenne muscular dystrophy community is in turmoil. The death of a second teenager from liver failure has sparked fear and anger. The teenager died after receiving a gene therapy called Elevidys. This therapy is from a company called Sarepta Therapeutics. The teenager's death has left famil

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A Baby's Life Saved by Tailored Gene Therapy

May 16, 2025

A major breakthrough in medicine has occurred. For the first time, doctors have used a custom-made CRISPR-based gene therapy to treat a baby's rare and deadly genetic condition. This isn't just any condition. It's a metabolic disorder that can kill up to half of the children who have it in their inf

health

Unlocking Africa's Medical Potential with Advanced Therapies

May 16, 2025

Africa faces a major challenge in healthcare. Many people suffer from diseases that could be treated with advanced therapies. These therapies, like cellular and gene treatments, have the power to change lives. They can improve survival rates, enhance quality of life, and reduce side effects. So, why

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Rare Genetic Conditions: The Hope and Hurdles of New Treatments

April 26, 2025

The world of genetic medicine is advancing rapidly, but not everyone is benefiting equally. People like Emily Kramer-Golinkoff, who has a rare form of cystic fibrosis, are often left behind. This disease, the most common fatal genetic condition in the U. S. , affects around 40, 000 Americans. Howeve