News

VEST Lowers Duration of Painful SCD Crises

Vascular electrical stimulation therapy (VEST), a form of local electrical stimulation applied to the skin, can lower the duration of painful vaso-occlusive crises (VOCs) in patients with sickle cell disease (SCD), according to data from a Phase 2 clinical trial. Based on these findings, study investigators argue that…

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…

Oxbryta Can Ease or Fully Heal Leg Ulcers, Trial Analysis Shows

Oxbryta (voxelotor) can help ease, or fully heal, painful leg ulcers in people with sickle cell disease (SCD), according to a post-hoc analysis of a Phase 3 trial. These promising findings support the launch of future trials investigating the potential benefits of Oxbryta — already conditionally approved…

Pandemic Won’t Stop Rare Disease Day on Feb. 28

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…