Pegvaliase for the treatment of phenylketonuria: Results of the phase 2 dose-finding studies with long-term follow-up

Barbara K. Burton; Nicola Longo; Jerry Vockley; Dorothy K. Grange; Cary O. Harding; Celeste Decker; Mingjin Li; Kelly Lau; Orli Rosen; Kevin Larimore; Janet Thomas

doi:10.1016/j.ymgme.2020.06.006

Pegvaliase for the treatment of phenylketonuria: Results of the phase 2 dose-finding studies with long-term follow-up

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2020.06.006 ◽

2020 ◽

Vol 130 (4) ◽

pp. 239-246

Author(s):

Barbara K. Burton ◽

Nicola Longo ◽

Jerry Vockley ◽

Dorothy K. Grange ◽

Cary O. Harding ◽

...

Keyword(s):

Dose Finding ◽

Phase 2 ◽

Long Term Follow Up

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A phase 2 study of sequential neoadjuvant chemotherapy with gemcitabine and doxorubicin followed by gemcitabine and cisplatin in patients with large or locally advanced operable breast cancer: results from long-term follow-up

Breast Cancer ◽

10.1007/s12282-012-0343-4 ◽

2012 ◽

Vol 20 (4) ◽

pp. 357-362 ◽

Cited By ~ 4

Author(s):

Pramod K. Julka ◽

Raju T. Chacko ◽

Shona Nag ◽

Rajinder Parshad ◽

Aravindan Nair ◽

...

Keyword(s):

Breast Cancer ◽

Neoadjuvant Chemotherapy ◽

Locally Advanced ◽

Operable Breast Cancer ◽

Phase 2 ◽

Phase 2 Study ◽

Long Term Follow Up ◽

Gemcitabine And Cisplatin

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Long-term follow-up of patients with poor-risk acute leukemia treated on a phase 2 trial undergoing intensified conditioning regimen prior to allogeneic hematopoietic cell transplantation

Leukemia & Lymphoma ◽

10.1080/10428194.2021.2012661 ◽

2021 ◽

pp. 1-7

Author(s):

Amandeep Salhotra ◽

Dongyun Yang ◽

Sally Mokhtari ◽

Susanta Hui ◽

Monzr M. Al Malki ◽

...

Keyword(s):

Acute Leukemia ◽

Cell Transplantation ◽

Hematopoietic Cell Transplantation ◽

Conditioning Regimen ◽

Hematopoietic Cell ◽

Phase 2 ◽

Phase 2 Trial ◽

Long Term Follow Up

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Editorial Comment on: Bladder Preservation in Selected Patients with Muscle-Invasive Bladder Cancer by Complete Transurethral Resection of the Bladder Plus Systemic Chemotherapy: Long-Term Follow-up of a Phase 2 Nonrandomized Comparative Trial with Radical Cystectomy

European Urology ◽

10.1016/j.eururo.2008.08.029 ◽

2009 ◽

Vol 55 (4) ◽

pp. 920-921 ◽

Cited By ~ 6

Author(s):

Harry W. Herr

Keyword(s):

Bladder Cancer ◽

Systemic Chemotherapy ◽

Comparative Trial ◽

Phase 2 ◽

Muscle Invasive Bladder Cancer ◽

Bladder Preservation ◽

Long Term Follow Up ◽

Muscle Invasive

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Lenalidomide/melphalan/dexamethasone in newly diagnosed patients with immunoglobulin light chain amyloidosis: results of a prospective phase 2 study with long-term follow up

Haematologica ◽

10.3324/haematol.2016.163246 ◽

2017 ◽

Vol 102 (8) ◽

pp. 1424-1431 ◽

Cited By ~ 20

Author(s):

Ute Hegenbart ◽

Tilmann Bochtler ◽

Axel Benner ◽

Natalia Becker ◽

Christoph Kimmich ◽

...

Keyword(s):

Light Chain ◽

Phase 2 ◽

Newly Diagnosed ◽

Immunoglobulin Light Chain ◽

Phase 2 Study ◽

Long Term Follow Up ◽

Prospective Phase ◽

Immunoglobulin Light Chain Amyloidosis

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Delineation of the Clinical Target Volume in Early-Stage Nasopharyngeal Carcinoma Treated With Intensity Modulated Radiation Therapy: Long-Term Follow-Up of a Phase 2 Study

International Journal of Radiation Oncology*Biology*Physics ◽

10.1016/j.ijrobp.2014.05.1616 ◽

2014 ◽

Vol 90 (1) ◽

pp. S529-S530 ◽

Cited By ~ 2

Author(s):

W. Xiao ◽

C. YanQing ◽

H. Fei ◽

L. LiXia ◽

Z. Chong

Keyword(s):

Clinical Target Volume ◽

Early Stage ◽

Intensity Modulated Radiation Therapy ◽

Target Volume ◽

Phase 2 ◽

Intensity Modulated ◽

Phase 2 Study ◽

Long Term Follow Up

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O4-08-07: Long-term follow up of Alzheimer's patients treated with bapineuzumab in phase 2

Alzheimer s & Dementia ◽

10.1016/j.jalz.2011.09.020 ◽

2011 ◽

Vol 7 ◽

pp. e7-e7

Author(s):

Stephen Salloway ◽

Reisa Sperling ◽

Lawrence Honig ◽

Kristen Morris ◽

Hsiao-Lan Wei ◽

...

Keyword(s):

Phase 2 ◽

Long Term Follow Up

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Efficacy and safety of erdafitinib in patients with locally advanced or metastatic urothelial carcinoma: long-term follow-up of a phase 2 study

The Lancet Oncology ◽

10.1016/s1470-2045(21)00660-4 ◽

2022 ◽

Author(s):

Arlene O Siefker-Radtke ◽

Andrea Necchi ◽

Se Hoon Park ◽

Jesús García-Donas ◽

Robert A Huddart ◽

...

Keyword(s):

Urothelial Carcinoma ◽

Locally Advanced ◽

Phase 2 ◽

Efficacy And Safety ◽

Phase 2 Study ◽

Long Term Follow Up ◽

Metastatic Urothelial Carcinoma

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Editorial Comment on: Bladder Preservation in Selected Patients with Muscle-Invasive Bladder Cancer by Complete Transurethral Resection of the Bladder Plus Systemic Chemotherapy: Long-Term Follow-up of a Phase 2 Nonrandomized Comparative Trial with Radical Cystectomy

European Urology ◽

10.1016/j.eururo.2008.08.028 ◽

2009 ◽

Vol 55 (4) ◽

pp. 919

Author(s):

J. Alfred Witjes

Keyword(s):

Bladder Cancer ◽

Systemic Chemotherapy ◽

Comparative Trial ◽

Phase 2 ◽

Muscle Invasive Bladder Cancer ◽

Bladder Preservation ◽

Long Term Follow Up ◽

Muscle Invasive

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Osimertinib in patients with T790M mutation‐positive, advanced non–small cell lung cancer: Long‐term follow‐up from a pooled analysis of 2 phase 2 studies

Cancer ◽

10.1002/cncr.31891 ◽

2018 ◽

Vol 125 (6) ◽

pp. 892-901 ◽

Cited By ~ 43

Author(s):

Myung‐Ju Ahn ◽

Chun‐Ming Tsai ◽

Frances A. Shepherd ◽

Lyudmila Bazhenova ◽

Lecia V. Sequist ◽

...

Keyword(s):

Lung Cancer ◽

Small Cell Lung Cancer ◽

Pooled Analysis ◽

Small Cell ◽

Phase 2 ◽

Small Cell Lung ◽

T790m Mutation ◽

Long Term Follow Up

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Rituximab in newly diagnosed stage IA nodular lymphocyte-predominant Hodgkin lymphoma: long-term follow-up of a phase 2 study from the German Hodgkin Study Group

Leukemia ◽

10.1038/s41375-019-0609-3 ◽

2019 ◽

Vol 34 (3) ◽

pp. 953-956 ◽

Cited By ~ 3

Author(s):

Dennis A. Eichenauer ◽

Annette Plütschow ◽

Michael Fuchs ◽

Sylvia Hartmann ◽

Martin-Leo Hansmann ◽

...

Keyword(s):

Hodgkin Lymphoma ◽

Study Group ◽

Phase 2 ◽

Newly Diagnosed ◽

German Hodgkin Study Group ◽

Phase 2 Study ◽

Long Term Follow Up ◽

Stage Ia

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