Intellia Therapeuticsは、5月12日に開催されたBank of Americaグローバル・ヘルスケア・カンファレンスにおいて、遺伝性血管性浮腫（HAE）を対象とした第III相HALO試験の速報データを発表しました。その結果、本疾患の患者に対して実質的な臨床的ベネフィットがあることが示されました。
同社の最高財務責任者（CFO）であるEdward Dulac氏は、セッションの中で今回の調査結果を「非常に勇気づけられるもの」と説明しました。すべての患者が有意義な臨床的改善を達成し、28週間の主要観察期間終了時点で62％の患者が発作を起こさず、かつ治療を必要としませんでした。発作発生率の低下は80％台後半に達しており、Dulac氏は、この成果は同分野におけるこれまでの報告と同等か、あるいはそれを上回るものであると述べています。
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A proof-of-concept randomized clinical trial published online May 20, 2026, in JAMA Psychiatry found signals that tocilizumab—an anti-inflammatory drug used for immune-mediated conditions including rheumatoid arthritis—may improve some symptoms in adults with moderate-to-severe depression who had a poor response to antidepressants and evidence of low-grade inflammation.
This Sunday marks World ALS Day with emphasis on new genetic treatments that have stabilized or restored motor functions in some patients.
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Syntara Limited held a shareholder webinar on April 29 to discuss FDA feedback on its proposed Phase IIb trial for amsulostat in myelofibrosis, along with a capital raise and quarterly report. CEO Gary Phillips highlighted the agreed path forward with regulators. The company lodged its Appendix 4C quarterly with the ASX.
Researchers at the University of Calgary are studying whether high doses of vitamin B3 (niacin) can improve outcomes for patients with newly diagnosed glioblastoma when added to standard treatment. An interim analysis of 24 patients found a higher-than-expected six‑month progression‑free survival rate, though investigators stress the results are preliminary and require ongoing safety monitoring.
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Pulse Biosciences outlined its progress with nanosecond pulsed field ablation technology during a presentation at the Bank of America Global Healthcare Conference on May 13.
Researchers at the University of Pennsylvania used artificial intelligence to analyze more than 400,000 Reddit posts about GLP-1 medications such as semaglutide and tirzepatide, identifying frequently discussed symptoms—including menstrual changes and temperature-related complaints—that may be less prominent in clinical trial reporting and drug labeling.
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A repurposed breast cancer drug called MDL-001 has shown promise in lab and animal studies against a range of viruses, including flu, covid-19, RSV and norovirus. Developed by California-based Model Medicines using AI, the pill targets a conserved enzyme domain in viruses. A clinical trial is planned for early next year.