In recognition of Rare Disease Day, the NeurologyLive® team offered an extensive update on the state of care and treatment for a wide range of rare neurological diseases, including Kleine-Levin syndrome, amyotrophic lateral sclerosis, cerebral palsy, Lennox-Gastaut syndrome, and more.
Ellie Harrington, lecturer in genetic counseling, provides answers for patients with ALS who might be unfamiliar with genetic testing and counseling.
The organization called attention to unintended harms of the current Medicare benefit structure and the need for the Center for Medicare & Medicaid Services to be more “patient-focused” in its assessments.
The combination drug of sodium phenylbutyrate–taurursodiol has been shown to functional decline in patients with amyotrophic lateral sclerosis compared with placebo in the phase 2/3 CENTAUR trial.
