comprehensive -WSS

comprehensive

author：comprehensive 2025-09-28 21:04:42 - browse：24634

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： How to save PrEP access — and even expand it
Next article： 'Investigate these claims': UFO transparency at center of House hearing

focus

hotspot

focus

WSS

Medicare drug price negotiation hearing pits pharma v Biden admin

He's 19 months old and has one of the world’s rarest diseases

Change Healthcare: What is it, why does its cyberattack matter?

Truvian presents data for its desktop blood

'Investigate these claims': UFO transparency at center of House hearing

Device maker Teleflex to acquire urology company for $600M

focus

comprehensive

hotspot

Duchenne breakthrough therapy leaves behind pioneering families

Dems pitch expanding Medicare power to negotiate drug prices

Medicare proposal to cover medical devices draws a mixed reaction

Medicare drug price negotiation continues, as pharma counteroffers

At least 13 dead in Texas as scorching temps continue

Biden's 'march

Virginia high school admissions case could be legal follow

Democrats decry new health bill for going easy on private equity

Malaria cases in Florida and Texas: Here’s what you need to know

What causes rare myocarditis in young men after Covid vaccines?

explore

Former reverend arrested for allegedly killing 8

What to watch for from Zogenix and Amicus clinical trials

Change Healthcare: What is it, why does its cyberattack matter?

Bronny James, LeBron's son, suffers cardiac arrest during basketball practice at USC

Ozempic has benefits for kidney disease as well as diabetes

Biogen says it will cut costs by $700 million, slash 1,000 jobs

focus

hotspot

focus

explore