News

Rare Disease Day Events Bring Awareness, Equity to Patients

Since 2008, Rare Disease Day — the last day of February — has brought together patients, caregivers, family members, friends, and advocates from around the world to raise awareness and improve equity for the more than 7,000 known rare diseases that affect more than 300 million people. In 2022, the…

Zilucoplan Safely Reduced gMG Symptoms in Phase 3 Trial

Zilucoplan, UCB’s experimental therapy, safely and effectively eases symptom severity and improves the quality of life for adults with generalized myasthenia gravis (gMG), while helping them complete daily activities, according to top-line data from the Phase 3 RAISE clinical trial. Based on these findings, indicating the trial met its…

Soleo Health Selected as US Distributor for Vyvgart for gMG

Soleo Health, a pharmacy specializing in infusion therapies, will serve as the U.S. distributor that will administer Vyvgart (efgartigimod), the newly approved treatment for people with generalized myasthenia gravis (gMG). Argenx, the therapy’s developer, selected Soleo Health as a limited distribution partner for Vyvgart, which was approved…

Japan Approves Vyvgart for Adults With Refractory gMG

Japan has approved Vyvgart (efgartigimod) to treat adults with generalized myasthenia gravis (gMG), the therapy’s developer, Argenx, announced. Vyvgart will be available to generalized MG patients, regardless of antibody status, who are refractory, which means they have failed to respond to steroids or nonsteroidal immunosuppressive therapies. The…

Model of Neuromuscular Junction May Help Evaluate MG Treatments

Scientists described a new cellular model of the neuromuscular junction that could be useful for diagnosing myasthenia gravis (MG) or developing new treatments for the condition. “New approaches for the treatment of neurodegenerative diseases are sorely needed, as decades of research have resulted in limited therapeutic advances. We hope…