AAV-Navβ1 gene therapy effectively reduces seizures and prolongs life in a mouse model of SCN1B-linked Dravet syndrome, ...

The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for vatiquinone for the treatment of children and adults living with Friedreich ataxia (FA).

We provide Severe Weather Warnings for potentially hazardous or dangerous weather that is not solely related to severe thunderstorms, tropical cyclones or bushfires. We provide Coastal Hazard Warnings ...

aDepartment of Pediatrics (Pediatric Neurology), Amalia Children's Hospital, Radboud University Medical Center, Nijmegen 6500, Netherlands ...

“In my clinical practice, I have seen the devastating impact that Friedreich’s ataxia has on patients ... Compromised mitochondrial function leads to chronic inflammation and vulnerability ...

While we experience many thunderstorms, more intense thunderstorms are referred to as severe thunderstorms. Severe thunderstorms can produce damaging wind gusts, large hail, tornadoes and heavy rain ...

The FDA has accepted PTC Therapeutics' NDA for vatiquinone, a potential treatment for Friedreich's ataxia, with a decision expected by August 19, 2025. PTC Therapeutics has announced that the FDA has ...

Chronic pharyngitis—also known as a chronic sore throat or persistent sore throat—is a sore throat that lasts several weeks or longer (sometimes 12 weeks or more). It can also occur as a sore throat ...

19, 2025. "We are excited to be one step closer to bringing an approved therapy to all patients with Friedreich's ataxia," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics.

WHEN a 44-year-old man began experiencing a common symptom experienced by millions, he decided to get checked out by medical ...

Millie, a modern maternity clinic, secured $12 million in Series A funding led by existing investors RH Capital, TMV, ...