hotspot -WSS

hotspot

author：focus 2025-10-02 22:40:09 - browse：194

Scanned cerebellum of a mouse brain, affected by Niemann-Pick Type C, shown in pink and teal — biotech coverage from STAT — The cerebellum of a mouse brain affected by Niemann-Pick Type C. Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health

About a decade ago, Tatiana Bremova-Ertl’s graduate adviser was studying an obscure, 1950s-era French vertigo drug, probing its effects on people with balance disorders, when she thought of another, very sick group of patients.

A graduate student and medical resident at the German Center for Vertigo and Balance Disorders, in Munich, Bremova-Ertl often saw patients with Niemann-Pick Type C, a rare, genetic disease that slowly kills neurons.

NPC has a range of manifestations. When symptoms appear in early childhood, it is often fatal before adulthood. When it manifests later, it can be milder. But it’s always degenerative and leads to a cluster of challenges: cognitive decline, difficulty with speech and swallowing, enlarged liver, low muscle tone, and, notably, difficulty with balance and muscle control. Researchers and a fervent group of parents were working to develop medicines, but little had yet worked.

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： Elizabeth Holmes on track for early release from her 11

WSS

Clinical trials giant ICON acquires HumanFirst

Biden launches a new push to limit health care costs

Why ADHD is under

Why getting cancer drugs just got harder for rural patients

Petition to FDA: Get the lead out of consumer hair dyes

Scientists unlock the key to scar

focus

hotspot

explore

Tori Bowie and the unacceptable rates of Black maternal death

RSV vaccines may be linked to small increased risk of developing GBS

The high cost of giving birth even with insurance

Bulgari apologizes to China for listing Taiwan as a country after online backlash

Indiana Supreme Court upholds abortion ban, says state constitution gives only limited protections

Obesity now affects over 1 billion people worldwide, study finds

As demand for Covid shots wanes, Moderna seeks its next success

Covid isolation guidance eased by the CDC

Medicare details structure of new drug price negotiation program

Scientists unlock the key to scar

focus

Charles Manson follower Leslie Van Houten released from prison after 53 years

How to address the problem of discarded donor organs

Threats against the LGBTQIA+ community intensifying: Department of Homeland Security

Biotech companies concerned about China biosecurity crackdown

Medical device lobby: We're tired of waiting on Medicare coverage

FDA does about

hotspot

hotspot

hotspot

entertainment