explore -WSS

explore

author：Wikipedia 2025-09-27 16:49:30 - browse：6364

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： Jerome Adams on the need for new antivirals for Covid
Next article： Does good food count as health care? Study aims to find out

hotspot

focus

WSS

O'Shea Sibley was killed after expressing queer joy by voguing, activists say

Weight loss drug Wegovy cuts risk of heart attacks in milestone trial

Some of the ways extreme heat will change life as we know it

U.K. considers world's first use of meningitis vaccine to address gonorrhea

GOP presidential candidates start declaring their loyalty

Pharma budget tightening trickles down to Doximity

explore

explore

knowledge

Drug repurposing or repositioning? The language matters

Weight loss drug costs may need more research, senator suggests

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

Where are patients getting their prescriptions for GLP

Virginia high school admissions case could be legal follow

Readout Newsletter: Bayer study halts, Carmot to IPO, and more

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

Surge of respiratory disease in China has likely explanation

The cancer drug shortage isn’t new — and neither are the solutions

Anthos stroke drug appears safer than common blood thinner

knowledge

Listen: Your guide to Wegovy's blockbuster heart study

Large hospital system sues Multiplan for low

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

Wegovy, Zepbound and weight loss drugs that preserve muscle

Readout Newsletter: What is Casgevy? Does Wegovy hurt muscle?

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

hotspot

leisure time

focus

leisure time