News

Study: Seizure Control May Benefit Development in Angelman Children

Most children with Angelman syndrome experience substantially delayed development, a new study from China highlights. Such developmental delays are especially pronounced in Angelman patients who experience seizures — which implies that seizure control may be beneficial for the development of children with the neurological disorder. The findings indicated…

FDA Acts to Support Development of Ionis Therapy ION582

The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease status to ION582, Ionis Pharmaceutical’s investigational treatment for Angelman syndrome. The announcement of the new FDA designations — both aimed at encouraging the therapy’s development — follows the launch of the…

Researchers Detail Abnormalities in Gaits of Mice With Angelman

Mice with Angelman syndrome (AS) show clear abnormalities in walking patterns, and assessing these patterns may be useful for measuring the efficacy of potential therapies, a new study indicates. “Gait analysis proved a reliable, translational assay that can accomplish within-subject lifespan development, regression, progression and/or decline without confounding test–retest…

Organizations Rally to Help Ukrainian Rare Disease Patients

A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ultimately brought the group of eight by car to temporary accommodations in neighboring Poland last month was physically and emotionally difficult,…