NealJWeinrebMD

PubMed

• The international cooperative Gaucher group (ICCG) Gaucher registry.
  Weinreb, N.> ;Best Practice & Research. Clinical Haematology. 2023 Dec 1
• 2 citations
  Ambroxol as Therapy for Gaucher Disease-Ambitious but Ambivalent.
  Neal J Weinreb, Ozlem Goker-Alpan> ;JAMA Network Open. 2023 Jun 1
• The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on Hughes et al. Switching between Enzyme Replacement Therapies and Substr...
  Pramod K Mistry, Priya S Kishnani, Manisha Balwani, Joel M Charrow, Judy Hull, Neal J Weinreb, Timothy M Cox> ;Journal of Clinical Medicine. 2023 May 4
• 10 citations
  Cancer risk and gammopathies in 2123 adults with Gaucher disease type 1 in the International Gaucher Group Gaucher Registry.
  Barry E Rosenbloom, Maria Domenica Cappellini, Neal J Weinreb, Marta Dragosky, Shoshana Revel-Vilk, Julie L Batista, Davorka Sekulic, Pramod K Mistry> ;American Journal of Hematology. 2022 Oct 1
• 2 citations
  Physiologically-Based Pharmacokinetic Model Development, Validation, and Application for Prediction of Eliglustat Drug-Drug Interactions.
  Siddhee A Sahasrabudhe, Shen Cheng, Mahmoud Al-Kofahi, Jeanine R Jarnes, Neal J Weinreb, Reena V Kartha> ;Clinical Pharmacology and Therapeutics. 2022 Dec 1
• 17 citations
  The diagnosis and management of Gaucher disease in pediatric patients: Where do we go from here?
  Neal J Weinreb, Ozlem Goker-Alpan, Priya S Kishnani, Nicola Longo, T Andrew Burrow, John A Bernat, Punita Gupta, Nadene Henderson, Helio Pedro, Carlos E Prada, Divya V...> ;Molecular Genetics and Metabolism. 2022 May 1
• 3 citations
  Safety and effectiveness of taliglucerase alfa in patients with Gaucher disease: an interim analysis of real-world data from a multinational drug registry (TALIAS).
  Lina Titievsky, Tilman Schuster, Ronnie Wang, Muhammad Younus, Andrew Palladino, Kabir Quazi, Michael P Wajnrajch, Betina Hernandez, Pamela S Becker, Neal J Weinreb, C...> ;Orphanet Journal of Rare Diseases. 2022 Apr 1
• 12 citations
  Gaucher disease - more than just a rare lipid storage disease.
  Jaehyeok Roh, Subbaya Subramanian, Neal J Weinreb, Reena V Kartha> ;Journal of Molecular Medicine. 2022 Apr 1
• 8 citations
  Screening, patient identification, evaluation, and treatment in patients with Gaucher disease: Results from a Delphi consensus.
  Priya S Kishnani, Walla Al-Hertani, Manisha Balwani, Özlem Göker-Alpan, Heather A Lau, Melissa Wasserstein, Neal J Weinreb, Gregory Grabowski> ;Molecular Genetics and Metabolism. 2022 Feb 1
• 9 citations
  Development and validation of Gaucher disease type 1 (GD1)-specific patient-reported outcome measures (PROMs) for clinical monitoring and for clinical trials.
  Deborah Elstein, Nadia Belmatoug, Patrick Deegan, Özlem Göker-Alpan, Derralynn A Hughes, Ida Vanessa D Schwartz, Neal Weinreb, Nicola Bonner, Charlotte Panter, Donna F...> ;Orphanet Journal of Rare Diseases. 2022 Jan 6
• 5 citations
  The International Collaborative Gaucher Group GRAF (Gaucher Risk Assessment for Fracture) score: a composite risk score for assessing adult fracture risk in imiglucera...
  Patrick Deegan, Aneal Khan, José Simon Camelo, Julie L. Batista, Neal J. Weinreb> ;Orphanet Journal of Rare Diseases. 2021 Feb 18
• 22 citations
  Gaucher disease type 1 patients from the ICGG Gaucher Registry sustain initial clinical improvements during twenty years of imiglucerase treatment.
  Neal J. Weinreb, José Simon Camelo, Joel Charrow, Monica R. McClain, Pramod K. Mistry, Nadia Belmatoug> ;Molecular Genetics and Metabolism. 2021 Jan 8
• 8 citations
  Patients with Gaucher disease display systemic oxidative stress dependent on therapy status
  Reena V. Kartha, Marcia R. Terluk, Roland Brown, Abigail Travis, Usha Mishra, Kyle Rudser, Heather Lau, Jeanine Jarnes, James C. Cloyd, Neal J. Weinreb> ;Molecular Genetics and Metabolism Reports. 2020 Dec 9
• 21 citations
  Gaucher disease and SARS-CoV-2 infection: Emerging management challenges.
  Pramod K. Mistry, Manisha Balwani, Deborah Barbouth, T. Andrew Burrow, Edward I. Ginns, Ozlem Goker-Alpan, Gregory A. Grabowski, Reena V. Kartha, Priya S. Kishnani, He...> ;Molecular Genetics and Metabolism. 2020 Jul 1
• 4 citations
  Neurochemical abnormalities in patients with type 1 Gaucher disease on standard of care therapy.
  Reena V. Kartha, James M. Joers, Marcia R. Terluk, Abigail Travis, Kyle Rudser, Paul J. Tuite, Neal J. Weinreb, Jeanine Jarnes, James C. Cloyd, Gülin Öz> ;Journal of Inherited Metabolic Disease. 2020 May 1
• 13 citations
  GBA1 mutations: Prospects for exosomal biomarkers in α-synuclein pathologies
  Parker H. Johnson, Neal J. Weinreb, James C. Cloyd, Paul J. Tuite, Reena V. Kartha> ;Molecular Genetics and Metabolism. 2020 Feb 1
• 65 citations
  Gaucher Disease in Bone: From Pathophysiology to Practice.
  Derralynn Hughes, P. Mikosch, Nadia Belmatoug, Francesca Carubbi, Timothy M. Cox, Ozlem Goker-Alpan, Andreas Kindmark, PramodK Mistry, Ludger Wilhelm Poll, Neal J. Wei...> ;Journal of Bone and Mineral Research. 2019 Jun 1
• 30 citations
  Presenting signs and patient co-variables in Gaucher disease: outcome of the Gaucher Earlier Diagnosis Consensus (GED-C) Delphi initiative
  Atul Mehta, David J. Kuter, Sam Salek, Nadia Belmatoug, Bruno Bembi, Jeremy R Bright, Stephan vom Dahl, Federica Deodato, Maja Di Rocco, Ozlem Goker-Alpan, Derralynn H...> ;Internal Medicine Journal. 2019 May 1
• 11 citations
  MGUS, lymphoplasmacytic malignancies, and Gaucher disease: the significance of the clinical association
  Neal J. Weinreb, Pramod K. Mistry, Barry E. Rosenbloom, Madhav V. Dhodapkar> ;Blood. 2018 May 31
• 29 citations
  Hepatocellular carcinoma in Gaucher disease: an international case series.
  Martine Regenboog, Laura van Dussen, Joanne Verheij, Neal J. Weinreb, David Santosa, Stephan vom Dahl, Dieter Häussinger, Meike N Müller, Ali Canbay, Miriam Rigoldi, A...> ;Journal of Inherited Metabolic Disease. 2018 Feb 8
• 27 citations
  Causes of death in 184 patients with type 1 Gaucher disease from the United States who were never treated with enzyme replacement therapy.
  Neal J. Weinreb, Deborah Barbouth, Robert E. Lee> ;Blood Cells, Molecules & Diseases. 2018 Feb 1
• 25 citations
  Transformation in pretreatment manifestations of Gaucher disease type 1 during two decades of alglucerase/imiglucerase enzyme replacement therapy in the International ...
  Pramod K. Mistry, Julie L. Batista, Hans C. Andersson, Manisha Balwani, Thomas A. Burrow, Joel Charrow, Paige Kaplan, Aneal Khan, Priya S. Kishnani, Edwin H. Kolodny, ...> ;American Journal of Hematology. 2017 Jul 7
• 1 citations
  Encore! Oral therapy for type 1 Gaucher disease
  Neal J. Weinreb> ;Blood. 2017 Apr 27
• 4 citations
  A new framework for evaluating the health impacts of treatment for Gaucher disease type 1
  Michael L. Ganz, Sean Stern, Alex Ward, Luba Nalysnyk, Martin Selzer, Alaa Hamed, Neal J. Weinreb> ;Orphanet Journal of Rare Diseases. 2017 Feb 20
• 89 citations
  Gaucher disease epidemiology and natural history: a comprehensive review of the literature
  Luba Nalysnyk, Philip Rotella, Jason C Simeone, Alaa Hamed, Neal J. Weinreb> ;Hematology. 2017 Feb 7
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Journal Articles

• Presenting Signs and Patient Co‐Variables in Gaucher Disease: Outcome of the Gaucher Early Diagnosis Consensus (GED‐C) Delphi Initiative  
  David J Kuter, Neal J Weinreb, Internal Medicine Journal