Authors: Claudia Chiriboga, Kathryn Swoboda, Basil Darras, Susan Iannaccone, Jacqueline Montes, Heather Allen, Rebecca Parad, Shanda Johnson, Darryl De Vivo, Daniel Norris, Katie Alexander, Frank Bennett, Kathie Bishop
Background/Significance: This first-in-human study was conducted to evaluate the safety, tolerability, and pharmacokinetics of escalating single intrathecal doses of ISIS-SMNRx in patients with SMA. ISIS-SMNRx is an antisense oligonucleotide molecule designed to alter splicing of SMN2 mRNA to increase production of functional SMN protein. The drug was found to be safe and well tolerated when given as a single dose. The results support continued development and further examination of ISIS-SMNRx in a longer, multiple-dose clinical study.
Session Information: S36.002, Anterior Horn: Clinical Trials
Presentation Time: Wednesday, March 20, 4:13 pm
Use of Oral Aprepitant for the Management of Nausea with Inpatient Intravenous Dihydroergotamine (DHE) Use
Authors: Denise E. Chou, Peter J. Goadsby
Background/Significance: This study assessed the efficacy and tolerability of oral aprepitant in controlling nausea associated with intravenous administration of dihydroergotamine (DHE) for medically refractory migraine. Oral aprepitant is used in the prevention of postoperative and chemotherapy-induced nausea and vomiting, but had not been studied in the prevention of DHE-related nausea. Oral aprepitant appears to be a novel, effective, and well-tolerated treatment for controlling nausea and vomiting related to intravenous DHE administration. Confirmation of these findings through further prospective studies would improve current intravenous DHE protocols for the treatment of refractory headache.
Session Information: S40.006, Headache: Clinical
Presentation Time: Wednesday, March 20, 5:05 pm
Weakness Is Independent of Fatigue in Diverse Ne
|Contact: Karin Eskenazi|
Columbia University Medical Center