PramodKMistryMDMB BS, FRCP, PhD

PubMed

• Liver Transplantation for Mahvash Disease, an Inborn Error of Metabolism.
  Pramod K Mistry, Guadalupe Garcia-Tsao> ;The New England Journal of Medicine. 2023 Nov 23
• 2 citations
  Advancing diagnosis and management of liver disease in adults through exome sequencing.
  Melanie Zheng, Aaron Hakim, Chigoziri Konkwo, Aimee M Deaton, Lucas D Ward, , Marina G Silveira, David N Assis, AnnMarie Liapakis, Ariel Jaffe, Z Gordon Jiang, Michael...> ;Ebiomedicine. 2023 Sep 1
• Osteonecrosis in Gaucher disease in the era of multiple therapies: Biomarker set for risk stratification from a tertiary referral center.
  Mohsen Basiri, Mohammad E Ghaffari, Jiapeng Ruan, Vagishwari Murugesan, Nathaniel Kleytman, Glenn Belinsky, Amir Akhavan, Andrew Lischuk, Lilu Guo, Katherine Klinger, ...> ;Elife. 2023 May 30
• 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
• 6 citations
  Venglustat combined with imiglucerase for neurological disease in adults with Gaucher disease type 3: the LEAP trial.
  Raphael Schiffmann, Timothy M Cox, Jean-François Dedieu, Sebastiaan J M Gaemers, Julia B Hennermann, Hiroyuki Ida, Eugen Mengel, Pascal Minini, Pramod Mistry, Petra B ...> ;Brain. 2023 Feb 13
• 1 citations
  Long-term effects of eliglustat on skeletal manifestations in clinical trials of patients with Gaucher disease type 1.
  Timothy M Cox, Joel Charrow, Elena Lukina, Pramod K Mistry, Meredith C Foster, M Judith Peterschmitt> ;Genetics in Medicine. 2023 Feb 1
• 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
• 10 citations
  Neuroinflammation in neuronopathic Gaucher disease: Role of microglia and NK cells, biomarkers, and response to substrate reduction therapy.
  Chandra Sekhar Boddupalli, Shiny Nair, Glenn Belinsky, Joseph Gans, Erin Teeple, Tri-Hung Nguyen, Sameet Mehta, Lilu Guo, Martin L Kramer, Jiapeng Ruan, Honggge Wang, ...> ;Elife. 2022 Aug 16
• 4 citations
  Therapies for lysosomal storage diseases: Principles, practice, and prospects for refinements based on evolving science.
  Gregory A Grabowski, Pramod K Mistry> ;Molecular Genetics and Metabolism. 2022 Jul 30
• 2 citations
  Transformative effect of a Humanitarian Program for individuals affected by rare diseases: building support systems and creating local expertise.
  I C Verma, A El-Beshlawy, A Tylki-Szymańska, A Martins, Y-L Duan, T Collin-Histed, M Schoneveld van der Linde, R Mansour, V C Dũng, Pramod K Mistry> ;Orphanet Journal of Rare Diseases. 2022 Apr 4
• Transjugular Intrahepatic Portosystemic Shunt for Refractory Ascites in Gaucher Disease.
  Kunal Adhyaru, Sherna Menezes, Pramod K Mistry, Aabha Nagral> ;Cureus. 2022 Apr 1
• 3 citations
  Long-read single molecule real-time (SMRT) sequencing of GBA1 locus in Gaucher disease national cohort from Argentina reveals high frequency of complex allele underlyi...
  Guillermo Drelichman, N. Fernandez Escobar, Barbara C. Soberon, Nora Basack, J. Frabasil, Andrea Schenone, Gabriel Aguilar, Maria Silvia Larroude, J. Knight, D. Zhao, ...> ;Molecular Genetics and Metabolism Reports. 2021 Nov 11
• 3 citations
  Miglustat Therapy for SCARB2-Associated Action Myoclonus-Renal Failure Syndrome.
  Imran H. Quraishi, Anna M. Szekely, Anushree C. Shirali, Pramod K. Mistry, Lawrence J. Hirsch> ;Neurology. Genetics. 2021 Jul 28
• 9 citations
  Incremental biomarker and clinical outcomes after switch from enzyme therapy to eliglustat substrate reduction therapy in Gaucher disease.
  Nathaniel Kleytman, Jiapeng Ruan, Audrey Ruan, Bailin Zhang, Vagishwari Murugesan, Haiqun Lin, Lilu Guo, Katherine W. Klinger, Pramod K. Mistry> ;Molecular Genetics and Metabolism Reports. 2021 Aug 28
• 4 citations
  The clinical spectrum of SARS-CoV-2 infection in Gaucher disease: Effect of both a pandemic and a rare disease that disrupts the immune system.
  Praveena Narayanan, Shiny Nair, Manisha Balwani, Maricar Malinis, Pramod K. Mistry> ;Molecular Genetics and Metabolism. 2021 Aug 13
• 19 citations
  Clinical outcomes after 4.5 years of eliglustat therapy for Gaucher disease type 1: Phase 3 ENGAGE trial final results.
  Pramod K. Mistry, Elena Lukina, Hadhami Ben Turkia, Suma P. Shankar, Hagit Baris Feldman, Marwan Ghosn, Atul Mehta, Seymour Packman, Heather Lau, Milan Petakov, Sarit ...> ;American Journal of Hematology. 2021 Sep 1
• 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
• 19 citations
  Gaucher disease and SARS-CoV-2 infection: Experience from 181 patients in New York
  Luca Fierro, Nora Nesheiwat, Hetanshi Naik, Praveena Narayanan, Pramod K. Mistry, Manisha Balwani> ;Molecular Genetics and Metabolism. 2021 Jan 1
• 25 citations
  Gaucher disease: Basic and translational science needs for more complete therapy and management.
  Gregory A. Grabowski, Armand H. Matheny Antommaria, Edwin H. Kolodny, Pramod K. Mistry> ;Molecular Genetics and Metabolism. 2021 Feb 1
• 12 citations
  Clinical relevance of endpoints in clinical trials for acid sphingomyelinase deficiency enzyme replacement therapy
  Simon Jones, Margaret M. McGovern, Olivier Lidove, Roberto Giugliani, Pramod K. Mistry, Carlo Dionisi-Vici, Maria Veronica Munoz-Rojas, L Nalysnyk, Alison D. Schecter,...> ;Molecular Genetics and Metabolism. 2020 Jun 24
• 12 citations
  Update on Alpha-1 Antitrypsin Deficiency in Liver Disease.
  Praveena Narayanan, Pramod K. Mistry> ;Clinical Liver Disease. 2020 Jun 1
• 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
• 18 citations
  Real-world effectiveness of eliglustat in treatment-naïve and switch patients enrolled in the International Collaborative Gaucher Group Gaucher Registry
  Pramod K. Mistry, Manisha Balwani, Joel Charrow, Priya S. Kishnani, Claus Niederau, Lisa H. Underhill, Monica R. McClain> ;American Journal of Hematology. 2020 May 21
• 19 citations
  Organic Solute Transporter Alpha Deficiency: A Disorder With Cholestasis, Liver Fibrosis, and Congenital Diarrhea.
  Emily Gao, Huma Arshad Cheema, Nadia K. Waheed, Iqra Mushtaq, Nihan Erden, Carol Nelson-Williams, Dhanpat Jain, Carol J. Soroka, James L. Boyer, Youssef Khalil, Peter ...> ;Hepatology. 2020 May 1
• 8 citations
  The Road to Biosimilars in Rare Diseases – Ongoing Lessons from Gaucher Disease
  Guillermo Drelichman, Gilberto Castañeda-Hernández, Muhlis Cem Ar, Marta Dragosky, Ricardo Sola Garcia, Howard Lee, Sergey Moiseev, Majid Naderi, Hanna Rosenbaum, Iren...> ;American Journal of Hematology. 2020 Mar 1
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Lectures

• D is for Diagnosis: Solving the Mystery of Rare Hematologic Disorders 
  60th American Society of Hematology Annual Meeting - 11/30/2018