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...
by Madaline Spencer and Kimberly Waller | Apr 1, 2026
Jack Johnson, Co-Founder and Executive Director of Fabry Support and Information Group (FSIG), discusses the organization’s 30 year anniversary. Fabry disease is a rare lysosomal storage disease characterized by a deficiency in the enzyme alpha-galactosidase...
by Madaline Spencer | Mar 31, 2026
The US Food and Drug Administration (FDA) has granted accelerated approval to Kresladi (marnetegragene autotemcel) for the treatment of pediatric patients with severe leukocyte adhesion deficiency-1 (LAD-1). The indication is for patients with biallelic variants in...
