The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). J. Clin. Med. 2022, 11, 5158

Pramod K. Mistry; Priya S. Kishnani; Manisha Balwani; Joel M. Charrow; Judy Hull; Neal J. Weinreb; Timothy M. Cox

doi:10.3390/jcm12093269

The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). J. Clin. Med. 2022, 11, 5158

Pramod K. Mistry^*, Priya S. Kishnani, Manisha Balwani, Joel M. Charrow, Judy Hull, Neal J. Weinreb, Timothy M. Cox

^*Corresponding author for this work

Pediatrics

Research output: Contribution to journal › Letter › peer-review

8 Scopus citations

Original language	English (US)
Article number	3269
Journal	Journal of Clinical Medicine
Volume	12
Issue number	9
DOIs	https://doi.org/10.3390/jcm12093269
State	Published - May 2023

Funding

Medical writing/editorial support for this article was paid for by Sanofi. P.K.M., P.S.K., M.B., J.M.C., N.J.W. and T.M.C. are principal investigators in Sanofi-sponsored clinical trials and/or members of regional advisory boards of the Sanofi-sponsored International Collaborative Gaucher Group Gaucher Registry, and have received research funding, educational grants, honoraria, consulting fees, and/or travel reimbursement from Sanofi. P.S.K. also reports serving on advisory boards for Amicus and Baebies; consulting fees from Amicus, AskBio, and Vertex; contracted research for Amicus and Valerion; honoraria from Amicus, AskBio, and Vertex; travel expenses from Amicus; and ownership interests in AskBio and Baebies. M.B. also reports participation on advisory boards for Takeda. N.J.W. also reports honoraria for consultancy or participation on advisory boards for Pfizer and Shire and fees or honoraria from speaking at the invitation of Pfizer and Shire. T.M.C. also reports consulting fees from AvroBio, Azafaros, Gain Therapeutics, Pfizer, and Takeda, and honoraria, travel reimbursement, and grant/research support from Shire Pharmaceuticals (now Takeda). J.H. is employed by Sanofi.

ASJC Scopus subject areas

General Medicine

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Mistry, P. K., Kishnani, P. S., Balwani, M., Charrow, J. M., Hull, J., Weinreb, N. J., & Cox, T. M. (2023). The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). J. Clin. Med. 2022, 11, 5158. Journal of Clinical Medicine, 12(9), Article 3269. https://doi.org/10.3390/jcm12093269

Mistry, Pramod K. ; Kishnani, Priya S. ; Balwani, Manisha et al. / The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease : Data from the Gaucher Outcome Survey (GOS). J. Clin. Med. 2022, 11, 5158. In: Journal of Clinical Medicine. 2023 ; Vol. 12, No. 9.

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title = "The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). J. Clin. Med. 2022, 11, 5158",

author = "Mistry, \{Pramod K.\} and Kishnani, \{Priya S.\} and Manisha Balwani and Charrow, \{Joel M.\} and Judy Hull and Weinreb, \{Neal J.\} and Cox, \{Timothy M.\}",

note = "Funding Information: Medical writing/editorial support for this article was paid for by Sanofi. Funding Information: P.K.M., P.S.K., M.B., J.M.C., N.J.W. and T.M.C. are principal investigators in Sanofi-sponsored clinical trials and/or members of regional advisory boards of the Sanofi-sponsored International Collaborative Gaucher Group Gaucher Registry, and have received research funding, educational grants, honoraria, consulting fees, and/or travel reimbursement from Sanofi. P.S.K. also reports serving on advisory boards for Amicus and Baebies; consulting fees from Amicus, AskBio, and Vertex; contracted research for Amicus and Valerion; honoraria from Amicus, AskBio, and Vertex; travel expenses from Amicus; and ownership interests in AskBio and Baebies. M.B. also reports participation on advisory boards for Takeda. N.J.W. also reports honoraria for consultancy or participation on advisory boards for Pfizer and Shire and fees or honoraria from speaking at the invitation of Pfizer and Shire. T.M.C. also reports consulting fees from AvroBio, Azafaros, Gain Therapeutics, Pfizer, and Takeda, and honoraria, travel reimbursement, and grant/research support from Shire Pharmaceuticals (now Takeda). J.H. is employed by Sanofi. ",

year = "2023",

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Mistry, PK, Kishnani, PS, Balwani, M, Charrow, JM, Hull, J, Weinreb, NJ & Cox, TM 2023, 'The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). J. Clin. Med. 2022, 11, 5158', Journal of Clinical Medicine, vol. 12, no. 9, 3269. https://doi.org/10.3390/jcm12093269

The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). J. Clin. Med. 2022, 11, 5158. / Mistry, Pramod K.; Kishnani, Priya S.; Balwani, Manisha et al.
In: Journal of Clinical Medicine, Vol. 12, No. 9, 3269, 05.2023.

Research output: Contribution to journal › Letter › peer-review

TY - JOUR

T1 - The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease

T2 - Data from the Gaucher Outcome Survey (GOS). J. Clin. Med. 2022, 11, 5158

AU - Mistry, Pramod K.

AU - Kishnani, Priya S.

AU - Balwani, Manisha

AU - Charrow, Joel M.

AU - Hull, Judy

AU - Weinreb, Neal J.

AU - Cox, Timothy M.

N1 - Funding Information: Medical writing/editorial support for this article was paid for by Sanofi. Funding Information: P.K.M., P.S.K., M.B., J.M.C., N.J.W. and T.M.C. are principal investigators in Sanofi-sponsored clinical trials and/or members of regional advisory boards of the Sanofi-sponsored International Collaborative Gaucher Group Gaucher Registry, and have received research funding, educational grants, honoraria, consulting fees, and/or travel reimbursement from Sanofi. P.S.K. also reports serving on advisory boards for Amicus and Baebies; consulting fees from Amicus, AskBio, and Vertex; contracted research for Amicus and Valerion; honoraria from Amicus, AskBio, and Vertex; travel expenses from Amicus; and ownership interests in AskBio and Baebies. M.B. also reports participation on advisory boards for Takeda. N.J.W. also reports honoraria for consultancy or participation on advisory boards for Pfizer and Shire and fees or honoraria from speaking at the invitation of Pfizer and Shire. T.M.C. also reports consulting fees from AvroBio, Azafaros, Gain Therapeutics, Pfizer, and Takeda, and honoraria, travel reimbursement, and grant/research support from Shire Pharmaceuticals (now Takeda). J.H. is employed by Sanofi.

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AN - SCOPUS:85159158287

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JO - Journal of Clinical Medicine

JF - Journal of Clinical Medicine

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Mistry PK, Kishnani PS, Balwani M, Charrow JM, Hull J, Weinreb NJ et al. The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). J. Clin. Med. 2022, 11, 5158. Journal of Clinical Medicine. 2023 May;12(9):3269. doi: 10.3390/jcm12093269

The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). J. Clin. Med. 2022, 11, 5158

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