News

FDA Gives Thumbs-up for Phase 2 Trial of Izokibep in AS Patients

The U.S. Food and Drug Administration (FDA) has cleared the start of a Phase 2 trial of izokibep in people with ankylosing spondylitis (AS), according to the therapy’s developers Affibody AB and Inmagene Biopharmaceuticals. “Obtaining FDA IND [investigational new drug] clearance is an important milestone for the izokibep…

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…

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…

Rinvoq Approved in EU for Two Types of Active Arthritis

AbbVie‘s Rinvoq (upadacitinib) has been approved in the European Union to treat active ankylosing spondylitis (AS) in adults who have responded inadequately to conventional therapy. The European Commission also approved Rinvoq to treat active psoriatic arthritis (PsA) in adults who have not responded to, or are…

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…