Risdiplam in Presymptomatic Spinal Muscular Atrophy
Infants up to 6 weeks of age with genetically diagnosed SMA who were treated with risdiplam before the development of clinical signs or symptoms appeared to have better functional and survival outcomes at 12 and 24 months than untreated infants in natural history studies. Larger, controlled studies with longer follow-up are needed to further understand the relative efficacy and safety of presymptomatic treatment of SMA with risdiplam.
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Ensitrelvir for Covid-19 Postexposure Prophylaxis in Household Contacts
Early initiation of a 5-day oral ensitrelvir regimen within 72 hours after symptom onset in an index patient significantly reduced the risk of developing Covid-19 among household contacts while maintaining a safety profile comparable to placebo. Clinical benefit was observed across major patient subgroups, including individuals at increased risk for severe disease, and treatment was associated with reduced household transmission. These findings support ensitrelvir as an effective postexposure prophylactic option for household contacts and suggest potential utility in other high-risk exposure settings where rapid outbreak control is needed.
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Pharmacogenomic-Informed Antidepressant Prescribing in Australian Primary Care
In this pragmatic double-blind randomized controlled trial conducted in Australian primary care, pharmacogenomic-informed antidepressant prescribing did not improve depressive symptoms compared with prescribing guided by national therapeutic guidelines. Clinical outcomes, medication tolerability, adherence, and quality of life were similar between groups, while cost-effectiveness analyses did not support routine implementation of pharmacogenomic testing in this setting. These findings suggest that broad pharmacogenomic-guided antidepressant prescribing in general practice does not provide additional clinical benefit over guideline-based management, although targeted evaluation in carefully selected patient populations may warrant further investigation.
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Low-Dose Buprenorphine Following Ketamine Treatment for Suicidal Ideation in Major Depressive Disorder
The Randomized Controlled Trial (RCT) provides the first evidence that low-dose buprenorphine can effectively extend the antisuicidal effects of a single intravenous ketamine infusion over 4 weeks in individuals with MDD. These findings offer a potentially scalable and safe therapeutic option for a population at risk of suicide.
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FDA Approves First Single-Dose Generic Treatment for Influenza
FDA approved the first generic single-dose baloxavir marboxil tablet for influenza in patients aged 5 years and above, ahead of the 20262027 flu season. Approved for both acute uncomplicated influenza treatment (within 48 hours of symptom onset) and post-exposure prophylaxis, this milestone may improve access, affordability, and ease of antiviral use in clinical practice.
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KCNQ2 Variants in Neonatal Epilepsy
KCNQ2-related neonatal epilepsy shows high early seizure controllability but divergent developmental trajectories that are associated with variant topology. Incorporating rapid genetic testing and topology-aware variant interpretation into neonatal care pathways may refine prognostication, guide counseling, and inform individualized therapy, while longitudinal imaging and functional studies are needed to strengthen mechanistic links and optimize outcomes.
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