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Manisha Balwani

Showing results (81-90 of 94) with videos related to

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American Journal of Hematology|June 23, 2021
Clinical outcomes after 4.5 years of eliglustat therapy for Gaucher disease type 1: Phase 3 ENGAGE trial final resultsPramod K Mistry, Elena Lukina, Hadhami Ben Turkia, et al.
JAMA|February 18, 2015
Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trialPramod K Mistry, Elena Lukina, Hadhami Ben Turkia, et al.
Movement Disorders : Official Journal of the Movement Disorder Society|March 3, 2023
Polygenic Parkinson's Disease Genetic Risk Score as Risk Modifier of Parkinsonism in Gaucher DiseaseCornelis Blauwendraat, Nahid Tayebi, Elizabeth Geena Woo, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|September 3, 2020
Evidence in the UK Biobank for the underdiagnosis of erythropoietic protoporphyriaAmy K Dickey, Corbin Quick, Sarah Ducamp, et al.
The New England Journal of Medicine|February 7, 2019
Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent PorphyriaEliane Sardh, Pauline Harper, Manisha Balwani, et al.
Molecular Medicine (Cambridge, Mass.)|June 11, 2015
Liver Transplantation for Acute Intermittent Porphyria: Biochemical and Pathologic Studies of the Explanted LiverMakiko Yasuda, Angelika L Erwin, Lawrence U Liu, et al.
Journal of the American Academy of Dermatology|August 30, 2022
Evidence-based consensus guidelines for the diagnosis and management of erythropoietic protoporphyria and X-linked protoporphyriaAmy K Dickey, Hetanshi Naik, Siobán B Keel, et al.
Journal of Hepatology|July 5, 2014
Sebelipase alfa over 52 weeks reduces serum transaminases, liver volume and improves serum lipids in patients with lysosomal acid lipase deficiencyVassili Valayannopoulos, Vera Malinova, Tomas Honzík, et al.
Hepatology Communications|February 19, 2025
Liver involvement in a large cohort of patients with erythropoietic protoporphyria or X-linked protoporphyriaCynthia Levy, Hetanshi Naik, Jessica Overbey, et al.
Molecular Genetics and Metabolism|July 3, 2026
Accessibility and harmonization of biochemical tests for diagnosis and monitoring of Porphyrias in the United States: Recommendations by members of the American Porphyrias Expert Collaborative (APEX)Karl E Anderson, Robert J Desnick, Herbert L Bonkovsky, et al.
Pageof 10

Showing results (81-90 of 94) with videos related to

Sort By:
Pageof 10
American Journal of Hematology|June 23, 2021
Clinical outcomes after 4.5 years of eliglustat therapy for Gaucher disease type 1: Phase 3 ENGAGE trial final resultsPramod K Mistry, Elena Lukina, Hadhami Ben Turkia, et al.
JAMA|February 18, 2015
Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trialPramod K Mistry, Elena Lukina, Hadhami Ben Turkia, et al.
Movement Disorders : Official Journal of the Movement Disorder Society|March 3, 2023
Polygenic Parkinson's Disease Genetic Risk Score as Risk Modifier of Parkinsonism in Gaucher DiseaseCornelis Blauwendraat, Nahid Tayebi, Elizabeth Geena Woo, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|September 3, 2020
Evidence in the UK Biobank for the underdiagnosis of erythropoietic protoporphyriaAmy K Dickey, Corbin Quick, Sarah Ducamp, et al.
The New England Journal of Medicine|February 7, 2019
Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent PorphyriaEliane Sardh, Pauline Harper, Manisha Balwani, et al.
Molecular Medicine (Cambridge, Mass.)|June 11, 2015
Liver Transplantation for Acute Intermittent Porphyria: Biochemical and Pathologic Studies of the Explanted LiverMakiko Yasuda, Angelika L Erwin, Lawrence U Liu, et al.
Journal of the American Academy of Dermatology|August 30, 2022
Evidence-based consensus guidelines for the diagnosis and management of erythropoietic protoporphyria and X-linked protoporphyriaAmy K Dickey, Hetanshi Naik, Siobán B Keel, et al.
Journal of Hepatology|July 5, 2014
Sebelipase alfa over 52 weeks reduces serum transaminases, liver volume and improves serum lipids in patients with lysosomal acid lipase deficiencyVassili Valayannopoulos, Vera Malinova, Tomas Honzík, et al.
Hepatology Communications|February 19, 2025
Liver involvement in a large cohort of patients with erythropoietic protoporphyria or X-linked protoporphyriaCynthia Levy, Hetanshi Naik, Jessica Overbey, et al.
Molecular Genetics and Metabolism|July 3, 2026
Accessibility and harmonization of biochemical tests for diagnosis and monitoring of Porphyrias in the United States: Recommendations by members of the American Porphyrias Expert Collaborative (APEX)Karl E Anderson, Robert J Desnick, Herbert L Bonkovsky, et al.
Pageof 10