Diseases & Diagnoses
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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...
Using a wearable technology (PKG) (Personal Kinetograph; Global Kinetics, Portsmouth, NH) cleared by the Food and Drug Administration (FDA), over 40,000 reports from individuals with Parkinson’s disease (PD) have been recorded and used by more than 200 Parkinson’s care centers. The PKG provides objective ambulatory measures of PD symptoms, including bradykinesia, dyskinesia, and tremor. Analysis of baseline data from the PKG showed that 54% of people reporting clinical measures with PKG had uncontrolled but likely treatable bradykinesia and 10% had uncontrolled, but likely...
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%...
Saif A. Bushnaq, MD; and Sunil A. Sheth, MD
Aniket Natekar, MD, MSc; Malya Sahu, BSc; Hsiangkuo Yuan, MD, PhD; and Stephanie Nahas, MD, MSEd, FAHS, FAAAN
Kate Onorato, MD; Carrie Dougherty, MD, FAHS; and Jessica Ailani, MD, FAHS