by Madaline Spencer | Apr 7, 2026
Miquel Vila-Perello, PhD, co-founder and CEO of SpliceBio, discusses an investigational dual adeno-associated virus (AAV) gene therapy in development for patients with Stargardt disease. Stargardt disease is a genetic eye disorder characterized by...
by Madaline Spencer | Apr 6, 2026
Richard A. Furie, MD, Chief of the Division of Rheumatology at Northwell Health, discusses nipocalimab for the treatment of systemic lupus erythematosus (SLE). SLE is an autoimmune disease characterized by inflammation in the connective tissues, leading to...
by Madaline Spencer | Apr 3, 2026
A study published in World Journal of Clinical Cases investigated the psychological aspects of rare autoimmune and ophthalmic diseases, such as mental well-being and everyday functioning. The goal of this study was to analyze descriptive case series reporting in 18...
by Madaline Spencer | Apr 2, 2026
Carlos A. Bacino, MD, Professor of Molecular and Human Genetics, Baylor College of Medicine and Texas Children’s Hospital, discusses the accelerated approval of Yuviwel (navepegritide) for patients with achondroplasia. Achondroplasia is a disorder of bone...
by Madaline Spencer and Kimberly Waller | Apr 2, 2026
The US Food and Drug Administration (FDA) has approved a high dose regimen of Spinraza (nusinersen) for the treatment of spinal muscular atrophy (SMA). SMA is a genetic neuromuscular disorder characterized by the loss of motor neurons, causing progressive muscle...
