Sarepta Warnings Came Long Before Its Standoff With US Regulators
After Sarepta's dramatic back-and-forth with the Food and Drug Administration, I started reaching out to patients and their families to see how they were feeling about the company's controversial gene therapies.
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Business Insider
6 hours ago
- Business Insider
Sarepta says eight-year-old death deemed unrelated to Elevidys
Sarepta (SRPT) said in a statement Friday night: 'Just before 6:00 p.m. ET today, the U.S. Food and Drug Administration issued a press release announcing an investigation into the death of an eight-year-old Duchenne muscular dystrophy patient who had received Elevidys gene therapy. The death of this patient was deemed unrelated to treatment with Elevidys…Sarepta reported this event to FDA on June 18, 2025, via the FDA's postmarketing electronic database, FAERS. At Sarepta, patient safety and well-being are always our top priority. We are committed to upholding the highest safety standards for all of our therapies, and do so in accordance with applicable law and commitment to full regulatory transparency. Elevidys is the only approved gene therapy for families and children devastated by Duchenne, a rare, progressive and ultimately fatal disease. We remain committed to working closely with the FDA to ensure that all decisions are grounded in science and the best interests of patients, considering the compelling need of these families to access disease-modifying therapy.' Elevate Your Investing Strategy:
New York Post
6 hours ago
- New York Post
Breakthrough as two FDA-approved drugs are found to reverse Alzheimer's — including restoring memory
In a stunning scientific discovery, researchers have found that a pair of drugs can not only slow down Alzheimer's disease but actually reverse it and restore memory in mice. And the best part of all? Both are already FDA-approved — albeit for cancer. Researchers first pinpointed how Alzheimer's disease scrambles gene activity in individual brain cells. 3 Researchers have found that a pair of drugs can not only slow down Alzheimer's disease but actually reverse it and restore memory in mice. Pixel-Shot – Using the Connectivity Map database of 1,300 FDA‑approved drugs, the researchers looked for medications that reverse Alzheimer's‑associated gene expression — landing on a shortlist of five, and zeroing in on two cancer drugs. In what one researcher called a 'mock clinical trial,' they then mined 1.4 million patients' medical records, finding that those who had taken letrozole or irinotecan for cancer were significantly less likely to develop Alzheimer's. When given together in an aggressive Alzheimer's mouse model, letrozole — used to treat certain types of breast cancer in postmenopausal women — and irinotecan — an anti-cancer medication used to treat colon cancer and small cell lung cancer — reversed disease‑related gene expression signatures, dissolved toxic tau protein clumps and prevented brain degeneration. Most importantly, they restored memory and learning in mice that had already developed severe symptoms. It's an exciting development for an illness that's notoriously tricky. 3 Letrozole — used to treat certain types of breast cancer in postmenopausal women — and irinotecan — an anti-cancer medication used to treat colon cancer and small cell lung cancer — reversed disease‑related gene expression signatures, dissolved toxic tau protein clumps and prevented brain degeneration. Eric Hood – 'Alzheimer's disease comes with complex changes to the brain, which has made it tough to study and treat, but our computational tools opened up the possibility of tackling the complexity directly,' Marina Sirota, the interim director of the UCSF Bakar Computational Health Sciences Institute, said in a statement. 'We're excited that our computational approach led us to a potential combination therapy for Alzheimer's based on existing FDA-approved medications.' 'Alzheimer's is likely the result of numerous alterations in many genes and proteins that, together, disrupt brain health,' said Yadong Huang, a professor of neurology and pathology at UCSF. 'This makes it very challenging for drug development — which traditionally produces one drug for a single gene or protein that drives disease.' 3 'Alzheimer's is likely the result of numerous alterations in many genes and proteins that, together, disrupt brain health,' said Yadong Huang, a professor of neurology and pathology at UCSF. yurakrasil – The findings were published in the journal Cell. Both drugs are already FDA‑approved for other uses, which could dramatically speed up the path to human trials. However, because they are cancer drugs, repurposing them may be complex and risky. This finding adds to a growing number of potential Alzheimer's treatments. A compound found in rosemary and sage — carnosic acid — has been shown to reverse memory loss and reduce brain inflammation in mice with Alzheimer's, bringing their cognitive function back to near-normal levels. A study from Stanford Medicine found that seniors who received the shingles vaccine were 20% less likely to develop dementia over seven years. And researchers at Penn State and Stanford University discovered that a certain cancer drug could restore memory and brain function in early stage Alzheimer's models.
Wall Street Journal
8 hours ago
- Wall Street Journal
Vinay Prasad Is a Bernie Sanders Acolyte in MAHA Drag
Meet Vinay Prasad, a young disciple of Bernie Sanders who ranks as one of the most powerful officials in the federal government. He determines whether patients get access to many life-saving medicines. Or not. Think of him as a one-man death panel. Dr. Prasad was named by Marty Makary, the Food and Drug Administration commissioner, as head of the agency's biologics division in May, and last month as its chief medical and scientific officer. More on why he was tapped to these posts later. But it isn't an exaggeration to say that Dr. Prasad wields more power than Anthony Fauci ever did at the National Institutes of Health.
