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Impel Neuropharma (Seattle, WA) is developing new therapeutic formulations for delivery of therapeutic compounds to the upper nasal cavity with their precision olfactory delivery (POD) device. The POD uses a propellant to deliver drugs to the upper nasal cavity, a new route of administration, which can provide higher bioavailability and rapid uptake vs traditional nasal sprays The POD device is easy to use, requiring only that the nasal tip of the device is inserted into the nostril and squeezed once to activate dose delivery. Coordination of breathing with activation is not required, and...
Post hoc analysis shows that participants who took 100 mg or 200 mg of lasmitidan had statistically significant increases in the rate of pain relief and freedom from the most bothersome symptom (MBS) (ie, nausea, photophobia, or phonophobia) (P < .001 starting as early as 30 minutes after administration. Participants who rated themselves as nonresponders, poor responders, or good responder to triptans did not have significant differences in their responses to 200 mg lasmiditan in any measure (freedom from pain at 2 hours, freedom from MBS, or pain relief). Participants who...
Over 7,500 individuals have been or are being treated with nusinersen (Spinraza; Biogen, Cambridge, MA). Across clinical trials and the real-world setting, there are now 7 years of data and experience showing that nusinersen has clinical efficacy and safety. In the NURTURE study (NCT02386553) in which presymptomatic infants likely to develop spinal muscular atrophy type one (SMA1) or type 2 (SMA2) were treated with nusinersen, children continue to develop and gain typical motor milestones. After 26 months of treatment, all infants could sit without support; 88% could walk with support and 77%...
Emmanuelle A. D. Schindler, MD, PhD; and Christopher Gottschalk, MD
Cynthia E. Armand, MD; Alina Masters-Israilov, MD; and Richard B. Lipton, MD
Lauren Doyle Strauss, DO, FAHS; and Scott Otallah, MD