Core Viewpoint - BioMarin Pharmaceutical plans to divest its gene therapy product, which was once anticipated to be a blockbuster treatment for a rare bleeding disorder [1] Company Summary - The gene therapy in question was previously expected to generate significant revenue as a leading treatment option for a specific rare bleeding disorder [1]

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 ...

Regulatory Approval - FDA 暂缓批准 Sarepta 将其有争议的基因疗法重新投放市场 [1] - FDA 需确认该疗法不会导致更多死亡案例后，才会批准 [1]

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 ...

Safety Concerns & Regulatory Impact - Serepta's gene therapy Elevidys linked to two patient deaths due to liver failure in 15 and 16-year-old boys with Duchenne muscular dystrophy [1][2] - FDA initially approved Elevidys for 4 and 5-year-olds who could still walk, later expanding the label to nearly all Duchenne patients, a decision that proved controversial due to limited evidence of efficacy in older patients [3] - Serepta has halted shipments of Elevidys for more advanced patients while exploring a new safety regimen [4] Financial Performance & Projections - Serepta is suspending its 2025 guidance and will update its full-year revenue outlook when it announces the second quarter results [4] - Prior analyst projections estimated Elevidys sales of $1.5 billion for the year, but these projections are now in serious doubt [4][5] Market Reaction & Stock Performance - Serepta's stock (SPTO) is down approximately 48% today and about 85% over the last year [5] - Safety concerns may cause eligible patients to reconsider treatment decisions [5]