Search research articles
Contact Us
Filters
Showing results (11-20 of 30) with videos related to
Page
of 3
Sort By:
JIMD Reports
|
January 17, 2025
Phenotypic variability and the gender paradox in the R363C variant of Fabry disease
Alison C Leslie, Jeanine Jarnes, Alia Ahmed, et al.
Pediatric Dermatology
|
October 6, 2023
Arteriovenous malformations as a presenting sign of PTEN hamartoma tumor syndrome: A case series
Morgan Dykman, Nishitha R Pillai, Kelsey Lenhart, et al.
International Journal of Neonatal Screening
|
March 27, 2026
Beyond Detection: Comparing State-Based Newborn Screening Methods for Effective Mucopolysaccharidosis I Diagnosis
Rithika Thampy, Nishitha R Pillai, Michael Evans, et al.
American Journal of Medical Genetics. Part A
|
March 3, 2025
Detecting the Difficult: An Intronic NPC1 Variant Hiding in Plain Sight
Caroline Gully Brown, Matthew Bower, Matthew Schomaker, et al.
Molecular Genetics and Metabolism
|
August 5, 2023
Hematopoietic cell transplantation for Mucopolysaccharidosis I in the presence of decreased cardiac function
Nishitha R Pillai, Sara A Elsbecker, Ashish O Gupta, et al.
Journal of Medical Economics
|
September 4, 2025
A retrospective cohort study of the economic burden of Pompe disease in patients treated with enzyme replacement therapy in the United States
Robert D Steiner, Tmirah Haselkorn, Nishitha R Pillai, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
March 17, 2025
Pegvaliase therapy for phenylketonuria: Real-world safety, efficacy, and medication access outcomes in a pharmacist-led pegvaliase program
Sofia Shrestha, Alicia L Zagel, Nishitha R Pillai, et al.
Journal of Neuromuscular Diseases
|
October 28, 2025
A retrospective cohort study describing the disease burden in patients with Pompe disease treated with enzyme replacement therapy in the United States
Nishitha R Pillai, Faryn Solomon, Robert D Steiner, et al.
European Journal of Medical Genetics
|
August 25, 2018
Novel deletion of 6p21.31p21.1 associated with laryngeal cleft, developmental delay, dysmorphic features and vascular anomaly
Nishitha R Pillai, Dana Marafi, Sonia A Monteiro, et al.
American Journal of Medical Genetics. Part A
|
July 11, 2019
Biallelic variants in COX4I1 associated with a novel phenotype resembling Leigh syndrome with developmental regression, intellectual disability, and seizures
Nishitha R Pillai, Noura S AlDhaheri, Rajarshi Ghosh, et al.
Page
of 3
Search research articles
Search
Showing results (11-20 of 30) with videos related to
Sort By:
Page
of 3
JIMD Reports
|
January 17, 2025
Phenotypic variability and the gender paradox in the R363C variant of Fabry disease
Alison C Leslie, Jeanine Jarnes, Alia Ahmed, et al.
Pediatric Dermatology
|
October 6, 2023
Arteriovenous malformations as a presenting sign of PTEN hamartoma tumor syndrome: A case series
Morgan Dykman, Nishitha R Pillai, Kelsey Lenhart, et al.
International Journal of Neonatal Screening
|
March 27, 2026
Beyond Detection: Comparing State-Based Newborn Screening Methods for Effective Mucopolysaccharidosis I Diagnosis
Rithika Thampy, Nishitha R Pillai, Michael Evans, et al.
American Journal of Medical Genetics. Part A
|
March 3, 2025
Detecting the Difficult: An Intronic NPC1 Variant Hiding in Plain Sight
Caroline Gully Brown, Matthew Bower, Matthew Schomaker, et al.
Molecular Genetics and Metabolism
|
August 5, 2023
Hematopoietic cell transplantation for Mucopolysaccharidosis I in the presence of decreased cardiac function
Nishitha R Pillai, Sara A Elsbecker, Ashish O Gupta, et al.
Journal of Medical Economics
|
September 4, 2025
A retrospective cohort study of the economic burden of Pompe disease in patients treated with enzyme replacement therapy in the United States
Robert D Steiner, Tmirah Haselkorn, Nishitha R Pillai, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
March 17, 2025
Pegvaliase therapy for phenylketonuria: Real-world safety, efficacy, and medication access outcomes in a pharmacist-led pegvaliase program
Sofia Shrestha, Alicia L Zagel, Nishitha R Pillai, et al.
Journal of Neuromuscular Diseases
|
October 28, 2025
A retrospective cohort study describing the disease burden in patients with Pompe disease treated with enzyme replacement therapy in the United States
Nishitha R Pillai, Faryn Solomon, Robert D Steiner, et al.
European Journal of Medical Genetics
|
August 25, 2018
Novel deletion of 6p21.31p21.1 associated with laryngeal cleft, developmental delay, dysmorphic features and vascular anomaly
Nishitha R Pillai, Dana Marafi, Sonia A Monteiro, et al.
American Journal of Medical Genetics. Part A
|
July 11, 2019
Biallelic variants in COX4I1 associated with a novel phenotype resembling Leigh syndrome with developmental regression, intellectual disability, and seizures
Nishitha R Pillai, Noura S AlDhaheri, Rajarshi Ghosh, et al.
Page
of 3