Diseases & Diagnoses
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The Food and Drug Administration (FDA) has scheduled an end-of-phase 2 clinical trial meeting for a sublingual formulation of racemic ketamine (Wafermine; iX Biopharma, Singapore) for treatment of moderate-to-severe acute pain. The therapy contains R and S isomers of ketamine in equal proportions In a phase 2b randomized placebo-controlled study (NCT03246971) participants experienced analgesic efficacy from ketamine with safety and tolerability for treatment of acute post-operative pain after abdominoplasty or bunionectomy. In pharmacokinetic studies (NCT01377831),...
Results from multiple trials of SMN1gene-replacement therapy (Zolgensma; Avexis, Bannockburn, IL) show promise for single-dose treatment of spinal muscular atrophy (SMA). In the STRIVE phase 3 study for treatment of spinal muscular atrophy, 22 infants under age 6 months, diagnosed genetically (biallelic SMN1 mutations/deletions, 2 SMN2 copies), were treated. Of the 15 babies treated more than 13.6 months or who discontinued the study before then, 13 (87%) survived without needing permanent ventilation, in contrast to the 25-50% survival rate for children with SMA when untreated....
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%...
Aniket Natekar, MD, MSc; Malya Sahu, BSc; Hsiangkuo Yuan, MD, PhD; and Stephanie Nahas, MD, MSEd, FAHS, FAAAN
Regina Krel, MD; and Paul G. Mathew, MD, DNBPAS, FAAN, FAHS
Lauren Doyle Strauss, DO, FAHS; and Scott Otallah, MD