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Durable Efficacy and Long-term Safety of AAV Gene Therapy in Severe Hemophilia B: 13-Year Follow-up Results

A single AAV gene therapy infusion for severe hemophilia B led to sustained factor IX expression, substantial reduction in bleeding and factor use, and no late safety concerns over 13 years.

Aug 1, 2025
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Zongertinib: A Promising Oral Targeted Therapy for Previously Treated HER2-Mutant Non–Small-Cell Lung Cancer

Zongertinib shows high response rates and manageable toxicity in HER2-mutant NSCLC after prior treatments, offering a new oral therapy option.

Aug 1, 2025Sign in to read
Clinical Updates

Efruxifermin for Compensated Liver Cirrhosis in MASH: Phase 2b Results and Clinical Implications

A phase 2b trial found that efruxifermin did not significantly improve fibrosis at 36 weeks in patients with compensated cirrhosis due to MASH, though longer-term benefit was suggested at 96 weeks.

Aug 1, 2025
Clinical Updates

Intra-arterial Tenecteplase After Thrombectomy for Acute Stroke: Interpreting the ANGEL-TNK Randomized Clinical Trial

The ANGEL-TNK trial found that intra-arterial tenecteplase after successful endovascular thrombectomy improved excellent neurological outcomes at 90 days without increasing bleeding or mortality, but secondary outcomes showed no significant

Jul 31, 2025
Clinical Updates

Intra-arterial Tenecteplase After Thrombectomy for Acute Stroke: Interpreting the ANGEL-TNK Randomized Clinical Trial

The ANGEL-TNK trial found that intra-arterial tenecteplase after successful endovascular thrombectomy improved excellent neurological outcomes at 90 days without increasing bleeding or mortality, but secondary outcomes showed no significant

Jul 31, 2025
Clinical Updates

High Tibial Osteotomy May Slow Knee Osteoarthritis Progression in Bowlegged Patients: Clinical Insights from a Randomized Trial

High tibial osteotomy (HTO) slows cartilage loss and improves pain and function in bowlegged patients with medial knee osteoarthritis compared to nonsurgical management, according to a new randomized trial.

Jul 30, 2025
Clinical Updates

FDA Fast-Tracks Rezpegaldesleukin for Severe Alopecia Areata: Promise for a Challenging Disease

The FDA has granted Fast Track status to rezpegaldesleukin (REZPEG), a novel biologic targeting regulatory T cells, for severe-to-very severe alopecia areata in patients aged ≥12 years. Phase 2b trial results are anticipated in December 202

Jul 30, 2025
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DM199 Shows Promise as a Potential Treatment for Preeclampsia

DM199 has demonstrated safety and efficacy in reducing blood pressure among preeclampsia patients during a Phase 2 trial, offering hope for improved maternal and fetal outcomes.

Jul 29, 2025
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