S. Sitaraman

发表

Regulatory strategies for rare diseases under current global regulatory statutes: a discussion with stakeholders

K. Romero, V. Sinha, F. Johnson, 2019, Orphanet Journal of Rare Diseases.

Novel quantitative method to evaluate globotriaosylceramide inclusions in renal peritubular capillaries by virtual microscopy in patients with fabry disease.

R. Colvin, L. Barisoni, S. Sitaraman, 2012, Archives of pathology & laboratory medicine.

Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial

B. Byrne, A. Pestronk, M. Tarnopolsky, 2021, The Lancet Neurology.

Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies

D. Lockhart, A. Mehta, R. Giugliani, 2012, Orphanet Journal of Rare Diseases.

Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase

D. Lockhart, J. Flanagan, C. Barlow, 2015, PloS one.

Evaluation of Globotriaosylceramide (GL-3) Accumulation in 18 Patients with Fabry Disease Nephropathy. Podocytes are More Severely Affected than Peritubular Capillaries

P. Fernhoff, A. Mehta, R. Giugliani, 2012 .

Migalastat HCl Reduces Globotriaosylsphingosine (Lyso-Gb3) in Fabry Transgenic Mice and in the Plasma of Fabry Patients

D. Lockhart, A. Mehta, R. Giugliani, 2013, PloS one.

Duvoglustat HCl Increases Systemic and Tissue Exposure of Active Acid α-Glucosidase in Pompe Patients Co-administered with Alglucosidase α

D. Lockhart, B. Byrne, J. Flanagan, 2017, Molecular therapy : the journal of the American Society of Gene Therapy.

Phase 3 study of migalastat HCl for Fabry disease: Stage 1 results

Salim Janmohamed, K. Nicholls, S. Sitaraman, 2013 .

The pharmacological chaperone 1-deoxygalactonojirimycin reduces tissue globotriaosylceramide levels in a mouse model of Fabry disease.

D. Lockhart, R. Desnick, Y. Lun, 2010, Molecular therapy : the journal of the American Society of Gene Therapy.

Interleukin-12 enhances clinical experimental autoimmune myasthenia gravis in susceptible but not resistant mice

D. Metzger, S. Sitaraman, R. J. Belloto, 2000, Journal of Neuroimmunology.

An open‐label study to determine the pharmacokinetics and safety of migalastat HCl in subjects with impaired renal function and healthy subjects with normal renal function

C. Barlow, P. Mudd, F. Johnson, 2015, Clinical pharmacology in drug development.

Use of the patient-reported outcomes measurement information system (PROMIS®) to assess late-onset Pompe disease severity

A. Mulberg, P. Kishnani, S. Sitaraman, 2020, Journal of Patient-Reported Outcomes.

Therapeutic Role of Pharmacological Chaperones in Lysosomal Storage Disorders: A Review of the Evidence and Informed Approach to Reclassification

S. Sitaraman, Jeffery P. Castelli, Ian Keyzor, 2023, Biomolecules.

Novel quantitative method to evaluate globotriaosylceramide inclusions in renal peritubular capillaries by virtual microscopy in patients with fabry disease.

R. Colvin, L. Barisoni, S. Sitaraman, 2012, Archives of pathology & laboratory medicine.

Pharmacokinetics and muscle distribution of AT2220, a pharmacological chaperone of acid-glucosidase, in healthy volunteers

S. Sitaraman, A. Bragat, P. Boudes, 2011 .