Le Lézard

Encoded Therapeutics Presents Positive Interim Efficacy Data from Initial Dose Levels of Phase 1/2 Trials Evaluating ETX101 Gene Therapy in Dravet Syndrome

Encoded Therapeutics Inc., a clinical-stage biotechnology company developing genetic medicines for severe neurological disorders, today announced positive interim results from its ongoing POLARIS ...

The ‘Cortisol Face’: Separating Hormonal Health from Viral Trends

We dissect Cortisol Face,' separating actual hormonal imbalance from general inflammation, and evaluate the answer and the ...

Ionis receives U.S. FDA Breakthrough Therapy designation for zilganersen for Alexander disease (AxD)

Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has granted ...
Le Lézard

Encoded Therapeutics to Present Interim Phase 1/2 Clinical Data on ETX101, the First One-time Gene Therapy in Development for Dravet Syndrome, at the 2025 American Epilepsy ...

Encoded Therapeutics Inc., a clinical-stage genetic medicines company, today announced upcoming presentations of interim clinical data from its ongoing POLARIS Phase 1/2 open-label trials evaluating ...

First Reported Alpha-Gal Syndrome Death, Meat Allergy From Tick Bite

The lone star tick along with the alpha-gal syndrome seems to be spreading in the U.S. Now there's been the first reported ...
Mathrubhumi English

Could uncontrolled laughter signal an underlying medical condition? Experts say yes

While laughter is generally considered beneficial, in rare cases it may indicate neurological or cardiac disorders, even in children.
WSKG

A red meat allergy caused by ticks killed a N.J. man. Here's what to know

Researchers say they believe they've documented the first known death from alpha-gal syndrome — a red meat allergy caused by tick bites. The findings, by researchers at the University of Virginia ...
Hosted on MSN

Ultragenyx Begins Dosing in Aurora Study for Angelman Syndrome

Take advantage of TipRanks Premium at 50% off! Unlock powerful investing tools, advanced data, and expert analyst insights to help you invest with confidence. Ultragenyx Pharmaceutical ( (RARE)) just ...
BioWorld

Study identifies ASO targeting UBE3A-ATS for Angelman syndrome

Angelman syndrome (AS) is a rare neurodevelopmental disorder caused by the loss of function of the maternally inherited ubiquitin E3 ligase UBE3A gene. AS is characterized by severe symptoms, ...
Benzinga.com

Ionis Pharmaceuticals To Expedite Development Of Rare Neurological Disease Candidate With FDA Breakthrough Status

On Tuesday, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to Ionis Pharmaceuticals Inc.’s (NASDAQ:IONS) ION582 for Angelman syndrome (AS), a rare neurological ...
Hosted on MSN

Broken Hill man starts 1,132km run to Newcastle fundraising for Angelman syndrome

Broken Hill man Mitch McKenny plans to run from the outback city to Newcastle in 29 days. He aims to raise awareness and funds for Angelman syndrome, a rare genetic disorder. Tradie 'fed up' as ...
WTNH

Cheshire car show supports kids with Angelman Syndrome

CHESHIRE, Conn. (WTNH) — The General Motors charity car show was held on Sunday in Cheshire, benefitting families with children diagnosed with Angelman Syndrome. Quinnipiac students create customized ...