focus -WSS

focus

author：knowledge 2025-09-27 16:48:38 - browse：45997

A doctor climbs up a red ladder to a vine shaped like a DNA — biotech coverage from STAT — Adobe

Every morning, Victoria Rasberry gets up before 6 a.m., walks to the bedroom down the hall, and helps her 8-year-old daughter Addi slide into a set of contraptions that squeeze and suck out the thick mucus constantly threatening to choke her airways closed. She feeds Addi breakfast through a stomach tube and makes sure she has the right mix of the 28 different medications required to keep her free of pain, infections, and seizures. At some point, Rasberry’s five other children will wake up and start to get ready for school, including her youngest, Oliver. At 3-and-a-half years old, he’s a typical toddler — always running and giggling and getting into things. You would never know that he and Addi were born with the same mutation lurking in their DNA, a genetic glitch that causes a fatal neurodegenerative disease called metachromatic leukodystrophy, or MLD.

“When Addi was this age she had lost the ability to speak and was already using a ventilator to sleep at night,” Rasberry said. “It’s amazing to see how well Ollie is doing in comparison. And it’s all because of the gene therapy.”

Three years ago, when Ollie was just 6 months old, he and Victoria traveled from their home in Texas to Milan, to receive a gene therapy treatment that adds a healthy version of the defective gene into patients’ own stem cells. At the time, traveling abroad for the therapy, which is manufactured by Orchard Therapeutics and was approved in Europe in 2021, was the only option for families stricken by MLD. But soon it will be available in the U.S., too.

Get unlimited access to award-winning journalism and exclusive events.

Subscribe Log In

Previous article： Malaria cases in Florida and Texas: Here’s what you need to know
Next article： Readout Newsletter: BridgeBio, Madrigal, and Silicon Valley Bank

focus

WSS

Why doctors need education on FDA device approval process

Flagship Pioneering launches a new biotech in two countries

eGenesis tests gene

Rep. Raul Ruiz on the physician shortage and more

EU regulator recommends expanding Mounjaro approval to obesity

Why doctors need education on FDA device approval process

focus

focus

hotspot

New air purifier tech could be key to improving indoor air quality

Readout Newsletter: Merck, J&J, and long Covid

Walensky prepares to leave CDC — and Congress — behind

J&J to emphasize cancer drugs, stop much of its vaccine research

Food as medicine: CMS rules hamper 'prescribing' of fruits, veggies

FDA clears AI tool for detecting skin cancer

23andMe had bad news about my health. I wish a person had delivered it

Saudi VC loops Novo Nordisk, biotech VCs into longevity incubator

Biden outlines 'new path' to provide student loan relief after Supreme Court rejection

UnitedHealth has 90,000 doctors — 10% of all physicians in U.S.

focus

Is mRNA technology the right fit for flu shots? Experts aren’t so sure

What don’t we know about an Alzheimer’s drug side effect?

Independent physicians launch a new lobbying group

Pediatric cancer treatments threatened by a new FDA policy

Readout LOUD podcast: Biotech layoffs, stock slump, 2024 preview

Independent physicians launch a new lobbying group

focus

hotspot

knowledge

focus