by Kimberly Waller | Apr 20, 2026
Prem Subramanian, MD, PhD, Chief of Neuro-Ophthalmology at the University of Colorado Anschutz, discusses Tepezza (teprotumumab-trbw) for the treatment of thyroid eye disease (TED). TED is a chronic endocrine, autoimmune disease characterized by...
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 Kimberly Waller | Feb 27, 2026
The US Food and Drug Administration (FDA) has approved Desmoda (desmopressin acetate) oral solution for the management of arginine vasopressin deficiency (AVP-D). AVP-D is a rare neuroendocrine disorder caused by the loss or impaired function of vasopressinergic...
by Kimberly Waller | Jul 15, 2025
Takeda has announced positive data from two phase 3 clinical trials of oveporexton in patients with narcolepsy type 1 (NT1). NT1 is a rare, chronic neurological disorder characterized by excessive daytime sleepiness (EDS) and cataplexy. It is caused by reduced levels...
