Ready, go. Chicago area dad William Smalls strives to care for his three sons equally, but says he and his wife Allison can't. Your insurance company is denying one son, correct.The same treatment your other son has received and seems to be thriving after receiving. Yes. It's baffling. It's the exact same diagnosis.That diagnosis, duchen musculardrophe or DMD, a genetic disorder that progressively attacks a person's muscles. Experts say most are in a wheelchair around 12 and don't live to see 30. Parents al ...

Longtime drug developer Suma Krishnan was 51 when she cofounded Krystal Biotech. Now the company has one gene therapy on the market and more in the works. #ForbesOver50 (Photo: Jamel Toppin for Forbes) https://t.co/eU154GZ8Kg https://t.co/fmDPWfCYnm ...

Shares of Sera Therapeutics up by about 36% in the pre-market. The company says that it will resume shipping Alvdus, that's a muscular disorder gene therapy to patients who can walk. Last week, the FDA recommended a pause in shipments because of serious safety concerns after three deaths of patients who were on this who were on serene therapies. This past weekend, there were reports of a death of another child on Avdus, but the death, the FDA now says, was unrelated to the gene therapy. In a statement, Surr ...

The FDA won’t sign off on Sarepta bringing its controversial gene therapy back to the market until the company can persuade US regulators that it won’t cause more deaths, according to an official familiar with the situation https://t.co/7gpkWSFdzG ...

There were warnings to the FDA about Sarepta before US regulators asked the company to halt shipments of its gene therapy https://t.co/ZZC0xgoUpS ...

For more on Soft's path ahead, Missouo healthcare strategist Jared Holes joins us here on set. Jared, great to have you with us. Um, the management had a call uh this morning and you were on that call.You talked to investors about the call. They highlighted that the the belief that they think that the ambulatory patient segment being treated with this drug could support profitability. But then there didn't seem to be real um comfort uh in in this event not happening in the in that group as well, right.I mea ...

We've got a number of downgrades as you might expect of Serepta today. This after the death of a second patient who received the company's gene therapy to treat Duchaine's muscular drophy. Angelica Peebles is here.She has more on the story for us. Angelica. Morning David.That's right. Surf is saying a 15-year-old who received the company's gene therapy. It's called Elevus dying from liver failure.And this is the second instance in just a matter of months. The other patient was a 16-year-old boy. And both of ...