by Madaline Spencer | Nov 15, 2024
X4 Pharmaceuticals announced positive results from their phase 2 clinical trial for mavorixafor in chronic neutropenia. Chronic neutropenic disorders are blood conditions characterized by low levels of neutrophils. These disorders are associated with...
by Madaline Spencer | Nov 15, 2024
Tuan Vu, MD, Professor of Neurology at the University of South Florida, discusses positive results in Vivacity-MG3 study evaulating the safety and efficacy of nipocalimab for myasthenia gravis (MG). MG is a chronic autoimmune neuromuscular disease...
by Madaline Spencer | Nov 14, 2024
Judith Thompson, Rare Disease Population Health Strategy Lead at UCB, discusses social determinants of health in rare diseases. As Ms. Thompson explains, social determinants of health are the conditions in which people live, work, and play. They impact...
by Madaline Spencer | Nov 13, 2024
Michael Hehir, MD, Professor of Neurology at the University of Vermont, discusses traditional versus novel therapies for myasthenia gravis (MG). MG is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles. The...
by Madaline Spencer | Nov 12, 2024
Joshua Todd, PhD, National Institute of Neurological Disorders and Stroke at the NIH, discusses the clinical development of adeno-associated virus (AAV)-based gene therapies for neuromuscular disorders. The development of new genetic therapies is essential to...
