News

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those were the findings of the…

Abnormal Flow of Sickled Cells Damages Blood Vessel Walls

Red blood cells in people with sickle cell disease (SCD) flow through blood vessels in a manner that damages the walls, which may explain blood vessel problems and increased inflammation in SCD, new research indicates. The study, “Flow-induced segregation and dynamics of red blood cells in sickle cell…

Lonza to Produce Aruvant’s ARU-1801 for Pivotal Trial

A recent manufacturing agreement between Aruvant Sciences and Lonza will advance the development of ARU-1801, an investigational gene therapy for the treatment of sickle cell disease (SCD). Under the agreement, Lonza will be responsible for producing Aruvant’s gene therapy for an upcoming pivotal clinical trial. The company already has…

NORD’s Caregiver Respite Program Continues Through Pandemic

Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for Rare Disorders (NORD)’s Rare Caregiver Respite Program may be a helpful resource. The program seeks to give a…