focus -WSS

focus

author：comprehensive 2025-10-02 00:52:46 - browse：3792

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： FDA approval of Sarepta Duchenne genetic therapy gives me hope
Next article： TB outbreak tied to bone grafts underscores testing shortcomings

WSS

Iran moves toward freeing 5 American citizens who had been imprisoned, in rare deal

Target recalls 2.2 million additional candles due to laceration, burn hazards

Beware reading your test results before your doctor does

The perfect storm of weather conditions that led to the devastating wildfires in Maui

The wait for US passports is creating travel purgatory and snarling summer plans

Trump, Nauta plead not guilty to new classified docs charges but De Oliveira still not arraigned

entertainment

focus

entertainment

Bright Health sells Medicare Advantage business to Molina

Iran moves toward freeing 5 American citizens who had been imprisoned, in rare deal

Bright Health sells Medicare Advantage business to Molina

Presidential candidate assassinated in Ecuador, 1 suspect dead and 6 arrested

Medicare details structure of new drug price negotiation program

The perfect storm of weather conditions that led to the devastating wildfires in Maui

Continuity nursing helped my family in our darkest PICU moments

Maui funeral service workers on devastation of fires: 'I'm in shock'

State Dept. review finds Biden bears some blame for Afghanistan failures

Use doctors trained outside the U.S. to ease the physician shortage

focus

Newly revealed memo details efforts to keep Trump in power following election loss

Undocumented workers face uncertainty as immigration law takes effect in Florida

Penis shrinkage, a side effect of prostate cancer surgery, is temporary

UpToDate has a racism problem: Dr. Stanley Goldfarb

Novo acquires Inversago for up to $1 billion

An innovative path out of biotech’s “valley of death”

focus

focus

entertainment

entertainment