Myrtelle’s candidate is rAAV-Olig001-ASPA, a gene therapy for Canavan disease (CD), a ... white matter of the brain to degenerate. People with VWM disease commonly have symptoms such as impaired ...

In April 2025, three people in Oregon developed Creutzfeldt-Jakob disease (CJD), a rare, fatal neurological condition that is ...

Paula Ritchie wasn’t dying, but under Canada’s new rules, she qualified for a medically assisted death. Was that kindness or ...

In a first-of-its-kind genome-wide association study (GWAS) researchers have discovered two genes, RNF144B and ENPP1, that ...

East Haven High teen faces physical therapy and homework to walk at graduation, inspiring hope and perseverance, after years of mobility issues.

Many rare disease patients do not receive a formal genetic diagnosis, but a European data-sharing project aimed to give them ...

A Spokane County man faces potential termination from his county job while he battles a rare and deadly skin disease that has kept him from working for several months.“I worry about my kids being ...

Delivered by a dedicated team of healthcare professionals trained in exercise assessment, prescription and progression, with experience of delivering patient-focused education on chronic respiratory ...