A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like…
Articles by Marisa Horak, MS
A patient with both Batten disease and Moyamoya disease — a condition where blood vessels in the skull…
Abeona Therapeutics and Taysha Gene Therapies have entered into agreements concerning ABO-202, an investigative gene therapy for…
The U.S. Food and Drug Administration (FDA) has awarded orphan drug designation and rare pediatric disease designation…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to a gene therapy being developed by …
The number of fluid-containing cellular compartments called vacuoles within immune cells could help to predict the severity of…
Two previously-unknown mutations in the CLN5 gene were reported to cause CLN5 disease, a form of late infantile…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to Amicus Therapeutics‘ AT-GTX-502, an investigational…
Polaryx Therapeutics has received approval from the U.S. Food and Drug Administration (FDA) to open a clinical trial…
An investigational gene therapy for late infantile (CLN2) Batten disease showed a good safety profile in a small clinical trial.