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...
by Kimberly Waller | Nov 26, 2024
Anne Marie Morse, DO, of Geisinger Health, discusses the U.S. Food and Drug Administration (FDA) approval of Lumryz (sodium oxybate) for the treatment of cataplexy or excessive daytime sleepiness in patients with narcolepsy ages 7 years and older. Narcolepsy is...
