by Kimberly Waller | May 7, 2026
Colin Lake, Head of Digital Business Transformation Neurology at UCB, discusses CareCompass, an online tool for caregivers of patients with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS). DS is the most severe of a group of conditions known as...
by Kimberly Waller | May 1, 2026
A study published in the Annals of the American Thoracic Society analyzed results from the ELEVATE-1 clinical trial testing the effects of rodatristat ethyl on daily activity in patients with pulmonary arterial hypertension (PAH). PAH is a rare condition affecting the...
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...
