Search Results - "Pipe SW"

Radionuclide synovectomy in patients with bleeding disorders: a review of malignancy and myeloproliferative neoplasms from the ATHNdataset.

Authors: Lim, M. Y. Cheng, D. Aschman, D. et al.

Source: Haemophilia. Mar2017, Vol. 23 Issue 2, pe160-e162. 3p.

Pain Reduction Following Eptacog Beta Treatment of Bleeding Episodes in Adolescents and Adults With Haemophilia A or B Complicated by Inhibitors.

Authors: Buckner TW Kessler C Castaman G et al.

Source: Haemophilia : the official journal of the World Federation of Hemophilia [Haemophilia] 2025 Jul; Vol. 31 (4), pp. 713-721. Date of Electronic Publication: 2025 Jun 19.

Publication Type: Journal Article; Randomized Controlled Trial

Journal Info: Publisher: Blackwell Science Country of Publication: England NLM ID: 9442916 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1365-2516 (Electronic) Linking ISSN: 13518216 NLM ISO Abbreviation: Haemophilia Subsets: MEDLINE

Global Comparative Antithrombin Field Study: Impact of Laboratory Assay Variability on the Assessment of Antithrombin Activity Measurement at Fitusiran Clinical Decision-Making Points.

Authors: Chhabra ES Sadeghi-Khomami A Liu M et al.

Source: Haemophilia : the official journal of the World Federation of Hemophilia [Haemophilia] 2025 May; Vol. 31 (3), pp. 566-574. Date of Electronic Publication: 2025 Apr 20.

Publication Type: Journal Article; Comparative Study

Journal Info: Publisher: Blackwell Science Country of Publication: England NLM ID: 9442916 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1365-2516 (Electronic) Linking ISSN: 13518216 NLM ISO Abbreviation: Haemophilia Subsets: MEDLINE

Gene Editing for Haemophilia-The Next Frontier.

Authors: Pinotti M Newby GA Selvaraj S et al.

Source: Haemophilia : the official journal of the World Federation of Hemophilia [Haemophilia] 2026 Apr 16. Date of Electronic Publication: 2026 Apr 16.

Publication Type: Journal Article

Journal Info: Publisher: Blackwell Science Country of Publication: England NLM ID: 9442916 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1365-2516 (Electronic) Linking ISSN: 13518216 NLM ISO Abbreviation: Haemophilia Subsets: MEDLINE

Durability of efficacy, safety, and quality of life 5 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A: final phase 3 GENEr8-1 trial results.

Authors: Leavitt AD Mahlangu J Raheja P et al.

Source: Research and practice in thrombosis and haemostasis [Res Pract Thromb Haemost] 2026 Mar 27; Vol. 10 (3), pp. 103416. Date of Electronic Publication: 2026 Mar 27 (Print Publication: 2026).

Publication Type: Journal Article; Clinical Trial, Phase III; Multicenter Study

Journal Info: Publisher: Elsevier Country of Publication: United States NLM ID: 101703775 Publication Model: eCollection Cited Medium: Internet ISSN: 2475-0379 (Electronic) Linking ISSN: 24750379 NLM ISO Abbreviation: Res Pract Thromb Haemost Subsets: MEDLINE

Redefining hemophilia management: treatment goals, nonfactor replacement therapies, and the role of fitusiran.

Authors: Pipe SW; Department of Hematology and Oncology, University of Michigan Medical School, 1500 E Medical Center Drive, MPB D4206, Ann Arbor, MI 48197. Email: ummdswp@med.umich.edu.

Source: The American journal of managed care [Am J Manag Care] 2026 Feb; Vol. 32 (2 Suppl), pp. S15-S24.

Publication Type: Journal Article

Journal Info: Publisher: Clinical Care Targeted Communications Group, LLC Country of Publication: United States NLM ID: 9613960 Publication Model: Print Cited Medium: Internet ISSN: 1936-2692 (Electronic) Linking ISSN: 10880224 NLM ISO Abbreviation: Am J Manag Care Subsets: MEDLINE

A comprehensive care pathway of gene therapy for hemophilia based on current guideline documents and summary of product characteristics: communication from the ISTH SSC working group on gene therapy.

Authors: Mussert CMA Miesbach W Chowdary P et al.

Source: Journal of thrombosis and haemostasis : JTH [J Thromb Haemost] 2026 Feb; Vol. 24 (2), pp. 747-765. Date of Electronic Publication: 2025 Oct 27.

Publication Type: Journal Article; Review; Practice Guideline

Journal Info: Publisher: Elsevier Country of Publication: England NLM ID: 101170508 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1538-7836 (Electronic) Linking ISSN: 15387836 NLM ISO Abbreviation: J Thromb Haemost Subsets: MEDLINE

Operationalising a Haemophilia Gene Editing Lexicon for Practical Use.

Authors: McKeown W Hermans C Unzu C et al.

Source: Haemophilia : the official journal of the World Federation of Hemophilia [Haemophilia] 2025 Mar; Vol. 31 (2), pp. 207-213. Date of Electronic Publication: 2025 Jan 27.

Publication Type: Journal Article

Journal Info: Publisher: Blackwell Science Country of Publication: England NLM ID: 9442916 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1365-2516 (Electronic) Linking ISSN: 13518216 NLM ISO Abbreviation: Haemophilia Subsets: MEDLINE

Outcomes and management of invasive procedures in participants with hemophilia A post gene therapy: a post hoc analysis of the GENEr8-1 phase III trial.

Authors: Quon DV Wang JD Wang M et al.

Source: Therapeutic advances in hematology [Ther Adv Hematol] 2024 Dec 19; Vol. 15, pp. 20406207241304645. Date of Electronic Publication: 2024 Dec 19 (Print Publication: 2024).

Publication Type: Journal Article

Journal Info: Publisher: Sage Country of Publication: England NLM ID: 101549589 Publication Model: eCollection Cited Medium: Print ISSN: 2040-6207 (Print) Linking ISSN: 20406207 NLM ISO Abbreviation: Ther Adv Hematol Subsets: PubMed not MEDLINE

Experience with trametinib in a pediatric patient with MAP2K1-related cervicofacial arteriovenous malformation.

Authors: Swaminathan N Smith JD Parmar HA et al.

Source: Pediatric blood & cancer [Pediatr Blood Cancer] 2024 Nov; Vol. 71 (11), pp. e31298. Date of Electronic Publication: 2024 Aug 27.

Publication Type: Case Reports; Letter

Journal Info: Publisher: John Wiley Country of Publication: United States NLM ID: 101186624 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1545-5017 (Electronic) Linking ISSN: 15455009 NLM ISO Abbreviation: Pediatr Blood Cancer Subsets: MEDLINE

A novel gene editing lexicon strategy for the haemophilia community: Research plan for development and preliminary results.

Authors: Hermans C Valentino LA Thornburg CD et al.

Source: Haemophilia : the official journal of the World Federation of Hemophilia [Haemophilia] 2024 Nov; Vol. 30 (6), pp. 1272-1280. Date of Electronic Publication: 2024 Oct 22.

Publication Type: Journal Article

Journal Info: Publisher: Blackwell Science Country of Publication: England NLM ID: 9442916 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1365-2516 (Electronic) Linking ISSN: 13518216 NLM ISO Abbreviation: Haemophilia Subsets: MEDLINE

Final Analysis of a Study of Etranacogene Dezaparvovec for Hemophilia B.

Authors: Pipe SW Miesbach W Recht M et al.

Corporate Authors: Pipe SW Miesbach W Recht M et al.

Source: The New England journal of medicine [N Engl J Med] 2026 Jan 29; Vol. 394 (5), pp. 463-474. Date of Electronic Publication: 2025 Dec 07.

Publication Type: Journal Article; Clinical Trial, Phase III; Multicenter Study

Journal Info: Publisher: Massachusetts Medical Society Country of Publication: United States NLM ID: 0255562 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1533-4406 (Electronic) Linking ISSN: 00284793 NLM ISO Abbreviation: N Engl J Med Subsets: MEDLINE

Etranacogene dezaparvovec in people with hemophilia B with preexisting adeno-associated virus 5 neutralizing antibodies: 4-year subgroup results from the HOPE-B trial.

Authors: Klamroth R Monahan PE Van der Valk P et al.

Source: Research and practice in thrombosis and haemostasis [Res Pract Thromb Haemost] 2026 Jan 19; Vol. 10 (1), pp. 103360. Date of Electronic Publication: 2026 Jan 19 (Print Publication: 2026).

Publication Type: Journal Article

Journal Info: Publisher: Elsevier Country of Publication: United States NLM ID: 101703775 Publication Model: eCollection Cited Medium: Internet ISSN: 2475-0379 (Electronic) Linking ISSN: 24750379 NLM ISO Abbreviation: Res Pract Thromb Haemost Subsets: In Process

Effect of etranacogene dezaparvovec on quality of life for severe and moderately severe haemophilia B participants: Results from the phase III HOPE-B trial 2 years after gene therapy.

Authors: Itzler R Buckner TW Leebeek FWG et al.

Source: Haemophilia : the official journal of the World Federation of Hemophilia [Haemophilia] 2024 May; Vol. 30 (3), pp. 709-719. Date of Electronic Publication: 2024 Mar 10.

Publication Type: Journal Article; Clinical Trial, Phase III

Journal Info: Publisher: Blackwell Science Country of Publication: England NLM ID: 9442916 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1365-2516 (Electronic) Linking ISSN: 13518216 NLM ISO Abbreviation: Haemophilia Subsets: MEDLINE

Natural history of preexisting AAV5 antibodies in adults with hemophilia B during the lead-in of the etranacogene dezaparvovec phase 3 study.

Authors: Klamroth R Recht M Key NS et al.

Source: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2025 Aug 18; Vol. 33 (3), pp. 101568. Date of Electronic Publication: 2025 Aug 18 (Print Publication: 2025).

Publication Type: Journal Article

Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101624857 Publication Model: eCollection Cited Medium: Print ISSN: 2329-0501 (Print) Linking ISSN: 23290501 NLM ISO Abbreviation: Mol Ther Methods Clin Dev Subsets: PubMed not MEDLINE

Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years.

Authors: von Drygalski A Gomez E Giermasz A et al.

Source: Blood advances [Blood Adv] 2025 Jul 22; Vol. 9 (14), pp. 3543-3552.

Publication Type: Journal Article; Clinical Trial, Phase II; Multicenter Study

Journal Info: Publisher: American Society of Hematology Country of Publication: United States NLM ID: 101698425 Publication Model: Print Cited Medium: Internet ISSN: 2473-9537 (Electronic) Linking ISSN: 24739529 NLM ISO Abbreviation: Blood Adv Subsets: MEDLINE

Safety and efficacy of a fitusiran antithrombin-based dose regimen in people with hemophilia A or B: the ATLAS-OLE study.

Authors: Young G Kavakli K Klamroth R et al.

Source: Blood [Blood] 2025 Jun 19; Vol. 145 (25), pp. 2966-2977.

Publication Type: Journal Article; Clinical Trial, Phase III; Multicenter Study

Journal Info: Publisher: Elsevier Country of Publication: United States NLM ID: 7603509 Publication Model: Print Cited Medium: Internet ISSN: 1528-0020 (Electronic) Linking ISSN: 00064971 NLM ISO Abbreviation: Blood Subsets: MEDLINE

Activated prothrombin complex concentrate in patients receiving emicizumab prophylaxis: from evidence to clinical practice.

Authors: Sidonio RF Jr Young G Escuriola Ettingshausen C et al.

Source: Research and practice in thrombosis and haemostasis [Res Pract Thromb Haemost] 2025 Jun 17; Vol. 9 (4), pp. 102926. Date of Electronic Publication: 2025 Jun 17 (Print Publication: 2025).

Publication Type: Journal Article; Review

Journal Info: Publisher: Elsevier Country of Publication: United States NLM ID: 101703775 Publication Model: eCollection Cited Medium: Internet ISSN: 2475-0379 (Electronic) Linking ISSN: 24750379 NLM ISO Abbreviation: Res Pract Thromb Haemost Subsets: PubMed not MEDLINE

Clinical perspective: Advancing hemophilia treatment through gene therapy approaches.

Authors: Thornburg CD Pipe SW Cantore A et al.

Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2025 Jun 04; Vol. 33 (6), pp. 2350-2362. Date of Electronic Publication: 2025 Apr 21.

Publication Type: Journal Article; Review

Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE

Guidelines for management of hemophilia-why, what, and how?

Authors: Srivastava A Mahlangu J Pipe SW

Source: Research and practice in thrombosis and haemostasis [Res Pract Thromb Haemost] 2025 May 02; Vol. 9 (4), pp. 102879. Date of Electronic Publication: 2025 May 02 (Print Publication: 2025).

Publication Type: Editorial

Journal Info: Publisher: Elsevier Country of Publication: United States NLM ID: 101703775 Publication Model: eCollection Cited Medium: Internet ISSN: 2475-0379 (Electronic) Linking ISSN: 24750379 NLM ISO Abbreviation: Res Pract Thromb Haemost Subsets: PubMed not MEDLINE