{"title":"The Cell and Gene Therapy Access Model - A Vision for Future Development.","authors":"Abe Sutton,Corinne Alberts,Andrew Xuan,Nicholas Minter,Abigale Sanft","doi":"10.1056/nejmp2513320","DOIUrl":"https://doi.org/10.1056/nejmp2513320","url":null,"abstract":"","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"82 1","pages":""},"PeriodicalIF":158.5,"publicationDate":"2026-03-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147374098","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Valuing Care Provided by Residents and Fellows - Toward Competency-Based Billing.","authors":"Abbas M Hassan,Jennifer F Waljee","doi":"10.1056/nejmp2514693","DOIUrl":"https://doi.org/10.1056/nejmp2514693","url":null,"abstract":"","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"58 1","pages":""},"PeriodicalIF":158.5,"publicationDate":"2026-03-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147374099","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Richard A Furie,Maria Dall'Era,Edward M Vital,Jay P Garg,Fedra Irazoque Palazuelos,Adolfina E Zuta Santillán,Jorge Ravelo-Hernández,Mittermayer B Santiago,Beatriz Zazueta Montiel,Stella Botha,Piotr Leszczyński,Viviane Angelina de Souza,Sandra A Sicsik,Luis Bellatin,Asokan Naidoo,Zahir Amoura,Maria Antonietta D'Agostino,Sunil Kumar,Biruh Workeneh,Julie Rae,Huiyan A Mao,Felix Erblang,Oliver Meier,Justine C Maller,Anca D Askanase,
BACKGROUNDObinutuzumab, a glycoengineered type II anti-CD20 monoclonal antibody, induces potent B-cell depletion and is approved for the treatment of active lupus nephritis. Its efficacy and safety in patients with active systemic lupus erythematosus (SLE) are yet to be determined.METHODSWe conducted a phase 3, multicenter, double-blind, placebo-controlled trial involving adults with active SLE but without proliferative or membranous lupus nephritis who were receiving standard therapy. Patients were randomly assigned in a 1:1 ratio to receive obinutuzumab (1000 mg) or placebo on day 1 and weeks 2, 24, and 26. In the prespecified analysis, the primary end point at week 52 was a response on the SLE Responder Index 4 (SRI-4), defined by a reduction from baseline of at least 4 points in the Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) score, no worsening of disease as assessed by the British Isles Lupus Assessment Group (BILAG) 2004 index and Physician's Global Assessment, and no intercurrent events (i.e., major concomitant-therapy violation, receipt of rescue medication, or early discontinuation of trial participation due to death, lack of efficacy, or adverse events).RESULTSOf 303 patients who underwent randomization, 151 were assigned to receive obinutuzumab and 152 to receive placebo. At week 52, an SRI-4 response was observed in 76.7% of the patients in the obinutuzumab group and in 53.5% of those in the placebo group (adjusted difference, 23.1 percentage points; 95% confidence interval [CI], 12.5 to 33.6; P<0.001). In an additional analysis whereby nonfatal intercurrent events did not affect response status, the respective percentages were 85.4% and 68.5% (adjusted difference, 16.8 percentage points; 95% CI, 7.1 to 26.4). Obinutuzumab was superior to placebo with respect to all key secondary end points: BILAG-based Composite Lupus Assessment response, sustained reduction in glucocorticoid dose, sustained SRI-4 response, SRI-6 response, and time to first BILAG-defined flare. Adverse events were reported in 88.7% of the patients in the obinutuzumab group and in 81.5% of those in the placebo group, and serious adverse events in 15.9% and 11.9%, respectively. One patient in the obinutuzumab group and 3 in the placebo group died during the double-blind period.CONCLUSIONSAmong adults with active SLE, treatment with obinutuzumab was superior to placebo with respect to the primary and all key secondary end points. (Funded by F. Hoffmann-La Roche; ALLEGORY ClinicalTrials.gov number, NCT04963296.).
{"title":"Efficacy and Safety of Obinutuzumab in Active Systemic Lupus Erythematosus.","authors":"Richard A Furie,Maria Dall'Era,Edward M Vital,Jay P Garg,Fedra Irazoque Palazuelos,Adolfina E Zuta Santillán,Jorge Ravelo-Hernández,Mittermayer B Santiago,Beatriz Zazueta Montiel,Stella Botha,Piotr Leszczyński,Viviane Angelina de Souza,Sandra A Sicsik,Luis Bellatin,Asokan Naidoo,Zahir Amoura,Maria Antonietta D'Agostino,Sunil Kumar,Biruh Workeneh,Julie Rae,Huiyan A Mao,Felix Erblang,Oliver Meier,Justine C Maller,Anca D Askanase, ","doi":"10.1056/nejmoa2516150","DOIUrl":"https://doi.org/10.1056/nejmoa2516150","url":null,"abstract":"BACKGROUNDObinutuzumab, a glycoengineered type II anti-CD20 monoclonal antibody, induces potent B-cell depletion and is approved for the treatment of active lupus nephritis. Its efficacy and safety in patients with active systemic lupus erythematosus (SLE) are yet to be determined.METHODSWe conducted a phase 3, multicenter, double-blind, placebo-controlled trial involving adults with active SLE but without proliferative or membranous lupus nephritis who were receiving standard therapy. Patients were randomly assigned in a 1:1 ratio to receive obinutuzumab (1000 mg) or placebo on day 1 and weeks 2, 24, and 26. In the prespecified analysis, the primary end point at week 52 was a response on the SLE Responder Index 4 (SRI-4), defined by a reduction from baseline of at least 4 points in the Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) score, no worsening of disease as assessed by the British Isles Lupus Assessment Group (BILAG) 2004 index and Physician's Global Assessment, and no intercurrent events (i.e., major concomitant-therapy violation, receipt of rescue medication, or early discontinuation of trial participation due to death, lack of efficacy, or adverse events).RESULTSOf 303 patients who underwent randomization, 151 were assigned to receive obinutuzumab and 152 to receive placebo. At week 52, an SRI-4 response was observed in 76.7% of the patients in the obinutuzumab group and in 53.5% of those in the placebo group (adjusted difference, 23.1 percentage points; 95% confidence interval [CI], 12.5 to 33.6; P<0.001). In an additional analysis whereby nonfatal intercurrent events did not affect response status, the respective percentages were 85.4% and 68.5% (adjusted difference, 16.8 percentage points; 95% CI, 7.1 to 26.4). Obinutuzumab was superior to placebo with respect to all key secondary end points: BILAG-based Composite Lupus Assessment response, sustained reduction in glucocorticoid dose, sustained SRI-4 response, SRI-6 response, and time to first BILAG-defined flare. Adverse events were reported in 88.7% of the patients in the obinutuzumab group and in 81.5% of those in the placebo group, and serious adverse events in 15.9% and 11.9%, respectively. One patient in the obinutuzumab group and 3 in the placebo group died during the double-blind period.CONCLUSIONSAmong adults with active SLE, treatment with obinutuzumab was superior to placebo with respect to the primary and all key secondary end points. (Funded by F. Hoffmann-La Roche; ALLEGORY ClinicalTrials.gov number, NCT04963296.).","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"250 1","pages":""},"PeriodicalIF":158.5,"publicationDate":"2026-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147359328","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Aspirin after PCI in Acute Coronary Syndromes.","authors":"Sofie Chin Ha Chan","doi":"10.1056/nejmc2518964","DOIUrl":"https://doi.org/10.1056/nejmc2518964","url":null,"abstract":"","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"14 1","pages":"1036"},"PeriodicalIF":158.5,"publicationDate":"2026-03-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147350588","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Hiddo J L Heerspink,Andreas L Birkenfeld,David Z I Cherney,Helen M Colhoun,Per-Henrik Groop,Linong Ji,Niels Jongs,Chantal Mathieu,Richard E Pratley,Sylvia E Rosas,Peter Rossing,Jay S Skyler,Katherine R Tuttle,Robert Lawatscheck,Meike Brinker,Markus F Scheerer,Julie Russell,Patrick Schloemer,Janet B McGill,
BACKGROUNDThe nonsteroidal mineralocorticoid receptor antagonist finerenone has been reported to improve kidney and cardiovascular outcomes in persons with type 2 diabetes and chronic kidney disease (CKD). The efficacy and safety of finerenone in persons with type 1 diabetes and CKD are unknown.METHODSWe conducted a phase 3 trial involving adults who had type 1 diabetes, CKD (estimated glomerular filtration rate [eGFR], 25 to <90 ml per minute per 1.73 m2 of body-surface area), and albuminuria (urinary albumin-to-creatinine ratio [with albumin measured in milligrams and creatinine measured in grams], 200 to <5000) and were receiving an angiotensin-converting-enzyme (ACE) inhibitor or an angiotensin-receptor blocker. Participants were randomly assigned to receive finerenone (10 or 20 mg per day, depending on the eGFR) or matching placebo. The primary outcome was the relative change in the urinary albumin-to-creatinine ratio over a period of 6 months.RESULTSA total of 242 participants underwent randomization. The median urinary albumin-to-creatinine ratio decreased from 574.6 at baseline to 373.5 at 6 months among all the participants assigned to receive finerenone and from 506.4 to 475.6 among those assigned to receive placebo. Over a period of 6 months, the urinary albumin-to-creatinine ratio decreased by 34% with finerenone (geometric mean ratio to baseline, 0.66; 95% confidence interval [CI], 0.60 to 0.73) and 12% with placebo (geometric mean ratio to baseline, 0.88; 95% CI, 0.79 to 0.98), which corresponded to a 25% greater reduction with finerenone than with placebo (geometric mean ratio for finerenone vs. placebo, 0.75; 95% CI, 0.65 to 0.87; P<0.001). The most common adverse event was hyperkalemia (in 12 participants [10.1%] with finerenone and in 4 [3.3%] with placebo); 2 participants (1.7%) discontinued finerenone because of hyperkalemia. At 6 months, the change in the eGFR was -5.6 ml per minute per 1.73 m2 with finerenone and -2.7 ml per minute per 1.73 m2 with placebo (difference, -2.9 ml per minute per 1.73 m2; 95% CI, -5.1 to -0.7); eGFR values approached baseline levels during the washout period.CONCLUSIONSIn adults with type 1 diabetes and CKD, finerenone resulted in a significantly greater decrease in the urinary albumin-to-creatinine ratio than placebo. (Funded by Bayer; FINE-ONE ClinicalTrials.gov number, NCT05901831.).
研究背景：非甾体类矿物皮质激素受体拮抗剂细烯酮已被报道可改善2型糖尿病和慢性肾脏疾病（CKD）患者的肾脏和心血管预后。芬烯酮对1型糖尿病和CKD患者的疗效和安全性尚不清楚。方法：我们进行了一项3期试验，纳入了患有1型糖尿病、CKD（估计肾小球滤过率[eGFR]，每分钟每1.73平方米体表面积25至<90 ml）和蛋白尿（尿白蛋白与肌酐比值[白蛋白以毫克计，肌酐以克计]，200至<5000）并接受血管紧张素转换酶（ACE）抑制剂或血管紧张素受体阻滞剂的成年人。参与者被随机分配接受细烯酮（每天10或20毫克，取决于eGFR）或匹配的安慰剂。主要结局是6个月期间尿白蛋白与肌酐比值的相对变化。结果242名受试者随机分组。在所有接受芬芬酮组的参与者中，尿白蛋白与肌酐比值的中位数从基线时的574.6下降到6个月时的373.5，在接受安慰剂组的参与者中从506.4下降到475.6。在6个月的时间里，芬尼酮组尿白蛋白与肌酐比值下降了34%（与基线的几何平均比值为0.66；95%可信区间[CI]为0.60至0.73），安慰剂组下降了12%（与基线的几何平均比值为0.88；95% CI为0.79至0.98），这相当于芬尼酮组比安慰剂组降低了25%（芬尼酮与安慰剂的几何平均比值为0.75；95% CI为0.65至0.87；P<0.001）。最常见的不良事件是高钾血症（芬尼酮组12例[10.1%]，安慰剂组4例[3.3%]）；2名参与者（1.7%）因高钾血症停用芬烯酮。6个月时，细芬烯酮组eGFR变化为-5.6 ml / min / 1.73 m2，安慰剂组为-2.7 ml / min / 1.73 m2（差异为-2.9 ml / min / 1.73 m2; 95% CI, -5.1至-0.7）；在洗脱期eGFR值接近基线水平。结论在成人1型糖尿病和CKD患者中，细芬烯酮显著降低尿白蛋白/肌酐比。（拜耳资助；FINE-ONE ClinicalTrials.gov编号：NCT05901831）。
{"title":"Finerenone in Type 1 Diabetes and Chronic Kidney Disease.","authors":"Hiddo J L Heerspink,Andreas L Birkenfeld,David Z I Cherney,Helen M Colhoun,Per-Henrik Groop,Linong Ji,Niels Jongs,Chantal Mathieu,Richard E Pratley,Sylvia E Rosas,Peter Rossing,Jay S Skyler,Katherine R Tuttle,Robert Lawatscheck,Meike Brinker,Markus F Scheerer,Julie Russell,Patrick Schloemer,Janet B McGill, ","doi":"10.1056/nejmoa2512854","DOIUrl":"https://doi.org/10.1056/nejmoa2512854","url":null,"abstract":"BACKGROUNDThe nonsteroidal mineralocorticoid receptor antagonist finerenone has been reported to improve kidney and cardiovascular outcomes in persons with type 2 diabetes and chronic kidney disease (CKD). The efficacy and safety of finerenone in persons with type 1 diabetes and CKD are unknown.METHODSWe conducted a phase 3 trial involving adults who had type 1 diabetes, CKD (estimated glomerular filtration rate [eGFR], 25 to <90 ml per minute per 1.73 m2 of body-surface area), and albuminuria (urinary albumin-to-creatinine ratio [with albumin measured in milligrams and creatinine measured in grams], 200 to <5000) and were receiving an angiotensin-converting-enzyme (ACE) inhibitor or an angiotensin-receptor blocker. Participants were randomly assigned to receive finerenone (10 or 20 mg per day, depending on the eGFR) or matching placebo. The primary outcome was the relative change in the urinary albumin-to-creatinine ratio over a period of 6 months.RESULTSA total of 242 participants underwent randomization. The median urinary albumin-to-creatinine ratio decreased from 574.6 at baseline to 373.5 at 6 months among all the participants assigned to receive finerenone and from 506.4 to 475.6 among those assigned to receive placebo. Over a period of 6 months, the urinary albumin-to-creatinine ratio decreased by 34% with finerenone (geometric mean ratio to baseline, 0.66; 95% confidence interval [CI], 0.60 to 0.73) and 12% with placebo (geometric mean ratio to baseline, 0.88; 95% CI, 0.79 to 0.98), which corresponded to a 25% greater reduction with finerenone than with placebo (geometric mean ratio for finerenone vs. placebo, 0.75; 95% CI, 0.65 to 0.87; P<0.001). The most common adverse event was hyperkalemia (in 12 participants [10.1%] with finerenone and in 4 [3.3%] with placebo); 2 participants (1.7%) discontinued finerenone because of hyperkalemia. At 6 months, the change in the eGFR was -5.6 ml per minute per 1.73 m2 with finerenone and -2.7 ml per minute per 1.73 m2 with placebo (difference, -2.9 ml per minute per 1.73 m2; 95% CI, -5.1 to -0.7); eGFR values approached baseline levels during the washout period.CONCLUSIONSIn adults with type 1 diabetes and CKD, finerenone resulted in a significantly greater decrease in the urinary albumin-to-creatinine ratio than placebo. (Funded by Bayer; FINE-ONE ClinicalTrials.gov number, NCT05901831.).","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"100 1","pages":"947-957"},"PeriodicalIF":158.5,"publicationDate":"2026-03-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147350590","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Linda Laux,Joseph Sullivan,M Scott Perry,Andreas Brunklaus,Archana Desurkar,John M Schreiber,Colin M Roberts,Kelly G Knupp,James W Wheless,Elaine C Wirrell,Pam Ventola,Fei Wang, Meena,Jessie Lynch,Kimberly A Parkerson,Barry Ticho,J Helen Cross,
BACKGROUNDDravet syndrome is a severe developmental and epileptic encephalopathy caused primarily by SCN1A haploinsufficiency. Risks of sudden unexpected death in epilepsy and cognitive deficits are higher among patients with this syndrome than in the general population with epilepsy. The effects of zorevunersen, an antisense oligonucleotide designed to up-regulate NaV1.1 sodium channels, in patients with Dravet syndrome are not known.METHODSWe enrolled patients 2 to 18 years of age with Dravet syndrome who were receiving standard antiseizure medications in two phase 1-2a, open-label, multicenter studies (MONARCH and ADMIRAL). Patients were included in either a single-ascending-dose cohort, in which zorevunersen (10 to 70 mg) was administered on day 1 only, or a multiple-ascending-dose cohort, in which zorevunersen (20 to 70 mg) was administered two or three times in a 3-month period. Patients eligible for rollover to the two open-label extension studies (SWALLOWTAIL and LONGWING) continued to receive zorevunersen (≤45 mg) every 4 months. The safety and pharmacokinetics of zorevunersen were assessed in the primary analysis; clinical effects were also evaluated.RESULTSA total of 81 patients were enrolled in the phase 1-2a studies. As of May 30, 2025, a total of 75 patients had entered the extension studies. Most adverse events were mild or moderate. The most common adverse event was post-lumbar puncture syndrome (in 25% of patients) in the phase 1-2a studies and was an elevated protein level in cerebrospinal fluid (in 45%) in the extension studies. One patient had suspected unexpected serious adverse reactions, 1 had an adverse event that led to study withdrawal, 2 died from sudden unexpected death in epilepsy, and 1 died from malnutrition. Patients who received 70 mg of zorevunersen (one, two, or three doses) in the phase 1-2a studies, followed by up to 45 mg in the extension studies, had a median change from baseline in convulsive-seizure frequency ranging from -58.82% to -90.91% across 1-month intervals during the first 20 months of the extension studies. The data supported improvements in overall clinical status, quality of life, and adaptive behavior with continued treatment for up to 36 months in the extension studies.CONCLUSIONSThe safety profile and initial clinical improvement support the continued development of zorevunersen as a potential disease-modifying treatment for Dravet syndrome. (Supported by Stoke Therapeutics; MONARCH and SWALLOWTAIL ClinicalTrials.gov numbers, NCT04442295 and NCT04740476, respectively; ADMIRAL and LONGWING ISRCTN Registry numbers, ISRCTN99651026 and ISRCTN12811235, respectively.).
{"title":"Zorevunersen in Children and Adolescents with Dravet Syndrome.","authors":"Linda Laux,Joseph Sullivan,M Scott Perry,Andreas Brunklaus,Archana Desurkar,John M Schreiber,Colin M Roberts,Kelly G Knupp,James W Wheless,Elaine C Wirrell,Pam Ventola,Fei Wang, Meena,Jessie Lynch,Kimberly A Parkerson,Barry Ticho,J Helen Cross, ","doi":"10.1056/nejmoa2506295","DOIUrl":"https://doi.org/10.1056/nejmoa2506295","url":null,"abstract":"BACKGROUNDDravet syndrome is a severe developmental and epileptic encephalopathy caused primarily by SCN1A haploinsufficiency. Risks of sudden unexpected death in epilepsy and cognitive deficits are higher among patients with this syndrome than in the general population with epilepsy. The effects of zorevunersen, an antisense oligonucleotide designed to up-regulate NaV1.1 sodium channels, in patients with Dravet syndrome are not known.METHODSWe enrolled patients 2 to 18 years of age with Dravet syndrome who were receiving standard antiseizure medications in two phase 1-2a, open-label, multicenter studies (MONARCH and ADMIRAL). Patients were included in either a single-ascending-dose cohort, in which zorevunersen (10 to 70 mg) was administered on day 1 only, or a multiple-ascending-dose cohort, in which zorevunersen (20 to 70 mg) was administered two or three times in a 3-month period. Patients eligible for rollover to the two open-label extension studies (SWALLOWTAIL and LONGWING) continued to receive zorevunersen (≤45 mg) every 4 months. The safety and pharmacokinetics of zorevunersen were assessed in the primary analysis; clinical effects were also evaluated.RESULTSA total of 81 patients were enrolled in the phase 1-2a studies. As of May 30, 2025, a total of 75 patients had entered the extension studies. Most adverse events were mild or moderate. The most common adverse event was post-lumbar puncture syndrome (in 25% of patients) in the phase 1-2a studies and was an elevated protein level in cerebrospinal fluid (in 45%) in the extension studies. One patient had suspected unexpected serious adverse reactions, 1 had an adverse event that led to study withdrawal, 2 died from sudden unexpected death in epilepsy, and 1 died from malnutrition. Patients who received 70 mg of zorevunersen (one, two, or three doses) in the phase 1-2a studies, followed by up to 45 mg in the extension studies, had a median change from baseline in convulsive-seizure frequency ranging from -58.82% to -90.91% across 1-month intervals during the first 20 months of the extension studies. The data supported improvements in overall clinical status, quality of life, and adaptive behavior with continued treatment for up to 36 months in the extension studies.CONCLUSIONSThe safety profile and initial clinical improvement support the continued development of zorevunersen as a potential disease-modifying treatment for Dravet syndrome. (Supported by Stoke Therapeutics; MONARCH and SWALLOWTAIL ClinicalTrials.gov numbers, NCT04442295 and NCT04740476, respectively; ADMIRAL and LONGWING ISRCTN Registry numbers, ISRCTN99651026 and ISRCTN12811235, respectively.).","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"42 1","pages":"969-982"},"PeriodicalIF":158.5,"publicationDate":"2026-03-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147350587","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Toward a Disease-Modifying Therapy for Dravet Syndrome.","authors":"Gemma L Carvill,Heather C Mefford","doi":"10.1056/nejme2515874","DOIUrl":"https://doi.org/10.1056/nejme2515874","url":null,"abstract":"","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"424 1","pages":"1022-1025"},"PeriodicalIF":158.5,"publicationDate":"2026-03-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147351023","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Shunting for Idiopathic Normal-Pressure Hydrocephalus.","authors":"Brian Walcott","doi":"10.1056/nejmc2600201","DOIUrl":"https://doi.org/10.1056/nejmc2600201","url":null,"abstract":"","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"31 1","pages":"1034"},"PeriodicalIF":158.5,"publicationDate":"2026-03-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147351088","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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