Asfotase alfa for infants and young children with hypophosphatasia: 7 year outcomes of a single-arm, open-label, phase 2 extension trial

Yearbook of Paediatric Endocrinology ◽

10.1530/ey.16.5.9 ◽

2019 ◽

Author(s):

Whyte MP ◽

Simmons JH ◽

Moseley S ◽

Fujita KP ◽

Bishop N ◽

...

Keyword(s):

Young Children ◽

Phase 2 ◽

Open Label ◽

Asfotase Alfa ◽

Open Label Phase ◽

Extension Trial ◽

Infants And Young Children

Asfotase alfa for infants and young children with hypophosphatasia: 7 year outcomes of a single-arm, open-label, phase 2 extension trial

The Lancet Diabetes & Endocrinology ◽

10.1016/s2213-8587(18)30307-3 ◽

2019 ◽

Vol 7 (2) ◽

pp. 93-105 ◽

Cited By ~ 27

Author(s):

Michael P Whyte ◽

Jill H Simmons ◽

Scott Moseley ◽

Kenji P Fujita ◽

Nicholas Bishop ◽

...

Keyword(s):

Young Children ◽

Phase 2 ◽

Open Label ◽

Asfotase Alfa ◽

Open Label Phase ◽

Extension Trial ◽

Infants And Young Children

Letter to the Editor: “Efficacy and Safety of Asfotase Alfa in Infants and Young Children With Hypophosphatasia: A Phase 2 Open-Label Study”

The Journal of Clinical Endocrinology & Metabolism ◽

10.1210/jc.2018-02413 ◽

2019 ◽

Vol 104 (8) ◽

pp. 3146-3147 ◽

Cited By ~ 1

Author(s):

Cheryl Rockman-Greenberg

Keyword(s):

Young Children ◽

Phase 2 ◽

Efficacy And Safety ◽

Open Label ◽

Open Label Study ◽

Label Study ◽

Asfotase Alfa ◽

Letter To The Editor ◽

Infants And Young Children

Efficacy and Safety of Asfotase Alfa in Infants and Young Children With Hypophosphatasia: A Phase 2 Open-Label Study

The Journal of Clinical Endocrinology & Metabolism ◽

10.1210/jc.2018-02335 ◽

2019 ◽

Vol 104 (7) ◽

pp. 2735-2747 ◽

Cited By ~ 17

Author(s):

Christine E Hofmann ◽

Paul Harmatz ◽

Jerry Vockley ◽

Wolfgang Högler ◽

Hideki Nakayama ◽

...

Keyword(s):

Young Children ◽

Phase 2 ◽

Efficacy And Safety ◽

Open Label ◽

Open Label Study ◽

Label Study ◽

Asfotase Alfa ◽

Infants And Young Children

Sustained efficacy and tolerability in infants and young children with life-threatening hypophosphatasia treated with asfotase alfa

Bone Abstracts ◽

10.1530/boneabs.3.pp363 ◽

2014 ◽

Cited By ~ 3

Author(s):

Michael Whyte ◽

Jill Simmons ◽

Richard Lutz ◽

Marc Vallee ◽

Agustin Melian ◽

...

Keyword(s):

Young Children ◽

Asfotase Alfa ◽

Sustained Efficacy ◽

Life Threatening ◽

Infants And Young Children

Improvement in bone manifestations and respiratory status in infants and young children with HPP treated with asfotase alfa: an update on the ENB-010-10 trial

Bone Abstracts ◽

10.1530/boneabs.4.oc18 ◽

2015 ◽

Cited By ~ 2

Author(s):

Christine Hofmann ◽

Cheryl Rockman-Greenberg ◽

Paul Harmatz ◽

Scott Moseley ◽

Tatjana Odrljin ◽

...

Keyword(s):

Young Children ◽

Asfotase Alfa ◽

Respiratory Status ◽

Infants And Young Children

Faculty Opinions recommendation of An open-label, phase 2 study evaluating the efficacy and safety of the anti-IGF-1R antibody cixutumumab in patients with previously treated advanced or metastatic soft-tissue sarcoma or Ewing family of tumours.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.718028323.793480222 ◽

2013 ◽

Author(s):

Richard Riedel

Keyword(s):

Soft Tissue ◽

Soft Tissue Sarcoma ◽

Phase 2 ◽

Efficacy And Safety ◽

Open Label ◽

Phase 2 Study ◽

Open Label Phase ◽

Metastatic Soft Tissue Sarcoma ◽

Previously Treated

Faculty Opinions recommendation of Atezolizumab versus docetaxel for patients with previously treated non-small-cell lung cancer (POPLAR): a multicentre, open-label, phase 2 randomised controlled trial.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.726215272.793539632 ◽

2017 ◽

Author(s):

Patrick Nana-Sinkam

Keyword(s):

Lung Cancer ◽

Randomised Controlled Trial ◽

Controlled Trial ◽

Small Cell ◽

Phase 2 ◽

Small Cell Lung ◽

Open Label ◽

Open Label Phase ◽

Previously Treated ◽

Randomised Controlled

Faculty Opinions recommendation of Luspatercept for the treatment of anaemia in patients with lower-risk myelodysplastic syndromes (PACE-MDS): a multicentre, open-label phase 2 dose-finding study with long-term extension study.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.730774358.793564974 ◽

2019 ◽

Author(s):

Anna Rita Migliaccio

Keyword(s):

Myelodysplastic Syndromes ◽

Lower Risk ◽

Dose Finding ◽

Extension Study ◽

Phase 2 ◽

Open Label ◽

Finding Study ◽

Open Label Phase ◽

Dose Finding Study

Faculty Opinions recommendation of Radiotherapy versus transoral robotic surgery and neck dissection for oropharyngeal squamous cell carcinoma (ORATOR): an open-label, phase 2, randomised trial.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.736408717.793563871 ◽

2019 ◽

Cited By ~ 1

Author(s):

Sandra Nuyts ◽

Sarah Deschuymer

Keyword(s):

Squamous Cell Carcinoma ◽

Robotic Surgery ◽

Cell Carcinoma ◽

Neck Dissection ◽

Squamous Cell ◽

Randomised Trial ◽

Transoral Robotic Surgery ◽

Phase 2 ◽

Open Label ◽

Open Label Phase

Dose-Adjusted EPOCH Regimen as First-Line Treatment for Non-Hodgkin's Lymphoma with Hemophagocytic Lymphohistiocytosis: A Single-Arm, Open-Label, Phase 2 Trial

SSRN Electronic Journal ◽

10.2139/ssrn.3304299 ◽

2018 ◽

Author(s):

Jin-Hua Liang ◽

Li Wang ◽

Hua-Yuan Zhu ◽

Jun Qian ◽

Hui Liao ◽

...

Keyword(s):

Hemophagocytic Lymphohistiocytosis ◽

Hodgkin's Lymphoma ◽

Line Treatment ◽

Phase 2 ◽

First Line ◽

Open Label ◽

Phase 2 Trial ◽

Non Hodgkin's Lymphoma ◽

Open Label Phase ◽

First Line Treatment