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Molecular Genetics and Metabolism Reports
|
December 18, 2020
Patients with Gaucher disease display systemic oxidative stress dependent on therapy status
Reena V Kartha, Marcia R Terluk, Roland Brown, et al.
British Journal of Clinical Pharmacology
|
February 3, 2016
A model-based approach to assess the exposure-response relationship of Lorenzo's oil in adrenoleukodystrophy
Mariam A Ahmed, Reena V Kartha, Richard C Brundage, et al.
Pediatric Nephrology (Berlin, Germany)
|
February 3, 2025
Model-informed repurposing of eliglustat for treatment and prophylaxis of Shiga toxin-producing Escherichia coli hemolytic-uremic syndrome (STEC-HUS) in children
David F G J Wolthuis, Jolien J M Freriksen, Mendy Ter Avest, et al.
Neurotherapeutics : the Journal of the American Society for Experimental Neurotherapeutics
|
April 8, 2026
Nervonic acid supplementation mitigates disease severity biomarkers in adrenoleukodystrophy
Chenxu Li, Kai Braaten, Ann B Moser, et al.
Journal of Psychiatry and Brain Science
|
May 26, 2021
Identifying Biological Signatures of N-Acetylcysteine for Non-Suicidal Self-Injury in Adolescents and Young Adults
Siddhee A Sahasrabudhe, Thanharat Silamongkol, Young Woo Park, et al.
Bjpsych Open
|
September 22, 2025
Examination of oxidative stress and glutamate as potential mechanisms of N-acetylcysteine in the treatment of non-suicidal self-injury in young people assigned female at birth: randomised trial
Victoria Papke, Bonnie Klimes-Dougan, Siddhee Anand Sahasrabudhe, et al.
Clinical Pharmacology and Therapeutics
|
August 16, 2024
Getting the Dose Right in Drug Development for Rare Diseases: Barriers and Enablers
Mariam A Ahmed, Rajesh Krishna, Noha Rayad, et al.
Orphanet Journal of Rare Diseases
|
October 24, 2021
Development of therapies for rare genetic disorders of GPX4: roadmap and opportunities
Dorian M Cheff, Alysson R Muotri, Brent R Stockwell, et al.
Molecular Genetics and Metabolism
|
May 31, 2020
Gaucher disease and SARS-CoV-2 infection: Emerging management challenges
Pramod Mistry, Manisha Balwani, Deborah Barbouth, et al.
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Search research articles
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Showing results (41-50 of 49) with videos related to
Sort By:
Page
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You have reached the last page of results.
This site can display upto 49 results.
Molecular Genetics and Metabolism Reports
|
December 18, 2020
Patients with Gaucher disease display systemic oxidative stress dependent on therapy status
Reena V Kartha, Marcia R Terluk, Roland Brown, et al.
British Journal of Clinical Pharmacology
|
February 3, 2016
A model-based approach to assess the exposure-response relationship of Lorenzo's oil in adrenoleukodystrophy
Mariam A Ahmed, Reena V Kartha, Richard C Brundage, et al.
Pediatric Nephrology (Berlin, Germany)
|
February 3, 2025
Model-informed repurposing of eliglustat for treatment and prophylaxis of Shiga toxin-producing Escherichia coli hemolytic-uremic syndrome (STEC-HUS) in children
David F G J Wolthuis, Jolien J M Freriksen, Mendy Ter Avest, et al.
Neurotherapeutics : the Journal of the American Society for Experimental Neurotherapeutics
|
April 8, 2026
Nervonic acid supplementation mitigates disease severity biomarkers in adrenoleukodystrophy
Chenxu Li, Kai Braaten, Ann B Moser, et al.
Journal of Psychiatry and Brain Science
|
May 26, 2021
Identifying Biological Signatures of N-Acetylcysteine for Non-Suicidal Self-Injury in Adolescents and Young Adults
Siddhee A Sahasrabudhe, Thanharat Silamongkol, Young Woo Park, et al.
Bjpsych Open
|
September 22, 2025
Examination of oxidative stress and glutamate as potential mechanisms of N-acetylcysteine in the treatment of non-suicidal self-injury in young people assigned female at birth: randomised trial
Victoria Papke, Bonnie Klimes-Dougan, Siddhee Anand Sahasrabudhe, et al.
Clinical Pharmacology and Therapeutics
|
August 16, 2024
Getting the Dose Right in Drug Development for Rare Diseases: Barriers and Enablers
Mariam A Ahmed, Rajesh Krishna, Noha Rayad, et al.
Orphanet Journal of Rare Diseases
|
October 24, 2021
Development of therapies for rare genetic disorders of GPX4: roadmap and opportunities
Dorian M Cheff, Alysson R Muotri, Brent R Stockwell, et al.
Molecular Genetics and Metabolism
|
May 31, 2020
Gaucher disease and SARS-CoV-2 infection: Emerging management challenges
Pramod Mistry, Manisha Balwani, Deborah Barbouth, et al.
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of 5