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Life-Changing Cystic Fibrosis Treatment Wins $3-Million Prize

Nature | Health & Medical Care

Life-Changing Cystic Fibrosis Treatment Wins $3-Million Prize

“The triple-drug combination Trikafta has given a new lease of life to 90% of people with cystic fibrosis, an inherited disorder that affects the lungs and other organs. Now, the trio of researchers who spearheaded its development has won one of this year’s US$3-million Breakthrough prizes — the most lucrative awards in science.”

From Nature.

Wall Street Journal | Science & Technology

The Devices That Will Read Your Brain—and Enhance It

“Right now, the market for devices that can read brain activity and translate it into actions is in its infancy. But, thanks in part to Elon Musk’s startup Neuralink, which is developing implantable brain-computer interfaces—or BCIs—that can record data from thousands of brain cells, investment and interest in these devices have soared in recent years.

New wearable devices designed to provide feedback during day-to-day activities build on implantable BCIs used for medical interventions, as well as decades of research into how the brain works. Efforts on implantable devices focus on restoring function. Applications aimed to allow communication and movement have been in development for decades to help paralyzed patients, and researchers have made major leaps with implantable devices in recent years, including giving voice back to the voiceless.”

From Wall Street Journal.

New York Times | Treatment of Animals

Could a Drug Give Your Pet More Dog Years?

“‘When you adopt a dog, you’re adopting future heartbreak,’ said Emilie Adams, a New Yorker who owns three Rhodesian Ridgebacks. ‘It’s worth it over time because you just have so much love between now and when they go. But their life spans are shorter than ours.’

In recent years, scientists have been chasing after drugs that might stave off this heartbreak by extending the lives of our canine companions. On Tuesday, the biotech company Loyal announced that it had moved one step closer to bringing one such drug to market. ‘The data you provided are sufficient to show that there is a reasonable expectation of effectiveness,’ an official at the U.S. Food and Drug Administration informed the company in a recent letter. (Loyal provided a copy of the letter to The Times.)

That means that the drug, which Loyal declined to identify for proprietary reasons, has met one of the requirements for ‘expanded conditional approval,’ a fast-tracked authorization for animal drugs that fulfill unmet health needs and require difficult clinical trials.”

From New York Times.

World Health Organization | Vaccination

Historic Step Towards Broader Vaccination against Malaria

“Shipments of the world’s first WHO-recommended malaria vaccine, RTS,S, have begun with 331 200 doses landing last night in Yaoundé, Cameroon…

A further 1.7 million doses of the RTS,S vaccine are expected to arrive in Burkina Faso, Liberia, Niger and Sierra Leone in the coming weeks, with additional African countries set to receive doses in the months ahead.”

From World Health Organization.

The Atlantic | Science & Technology

The CRISPR Era Is Here

“Four years ago, [Victoria Gray] joined a groundbreaking clinical trial that would change her life. She became the first sickle-cell patient to be treated with the gene-editing technology CRISPR—and one of the first humans to be treated with CRISPR, period. CRISPR at that point had been hugely hyped, but had largely been used only to tinker with cells in a lab. When Gray got her experimental infusion, scientists did not know whether it would cure her disease or go terribly awry inside her. The therapy worked—better than anyone dared to hope. With her gene-edited cells, Gray now lives virtually symptom-free. Twenty-nine of 30 eligible patients in the trial went from multiple pain crises every year to zero in 12 months following treatment.”

From The Atlantic.