explore -WSS

explore

author：focus 2025-09-30 21:57:09 - browse：46

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： New air purifier tech could be key to improving indoor air quality
Next article： Readout Newsletter: Ironwood, Pfizer, FogPharma updates

entertainment

WSS

Covid isolation guidance eased by the CDC

Covid isolation guidance eased by the CDC

Kristina Burow of ARCH Venture Partners shares her next big bets STAT

Scientists unlock the key to scar

Why getting cancer drugs just got harder for rural patients

Biotech companies concerned about China biosecurity crackdown

hotspot

explore

explore

Drug repurposing or repositioning? The language matters

Obesity now affects over 1 billion people worldwide, study finds

After affirmative action ruling, medical educators look to 'holistic review'

Congress gives doctors Medicare pay bump

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

Abortion pill mifepristone to soon be dispensed at CVS, Walgreens

ChatGPT in medicine: STAT answers readers' burning questions

Kristina Burow of ARCH Venture Partners shares her next big bets STAT

What does generative AI mean for health care? We asked experts

Readout LOUD Podcast: Legal insider trading, booming biotech stocks

focus

An inside look at PIPEs, biotech's buzzy new financing tool

Pfizer highlights cancer drugs it thinks could reignite investor interest

Scientists unlock the key to scar

Readout LOUD Podcast: Legal insider trading, booming biotech stocks

An inside look at PIPEs, biotech's buzzy new financing tool

An inside look at PIPEs, biotech's buzzy new financing tool

hotspot

focus

entertainment

Wikipedia