Omar S. Khwaja

Neurology

300 Longwood Ave
Boston, MA 02115

Last Updated: 09/26/2026

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Found 44 publications

Author Response: Safety of AADC Gene Therapy for Moderately Advanced Parkinson Disease: Three-Year Outcomes From the PD-1101 Trial.

Author Response: Safety of AADC Gene Therapy for Moderately Advanced Parkinson Disease: Three-Year Outcomes From the PD-1101 Trial.

Journal: Neurology

Published: August 08, 2022

Author Response: Safety of AADC Gene Therapy for Moderately Advanced Parkinson Disease: Three-Year Outcomes From the PD-1101 Trial.

Author Response: Safety of AADC Gene Therapy for Moderately Advanced Parkinson Disease: Three-Year Outcomes From the PD-1101 Trial.

Journal: Neurology

Published: August 08, 2022

Convergent cerebrospinal fluid proteomes and metabolic ontologies in humans and animal models of Rett syndrome.

Convergent cerebrospinal fluid proteomes and metabolic ontologies in humans and animal models of Rett syndrome.

Journal: iScience

Published: June 17, 2022

A randomized, double-blind, placebo-controlled phase II trial to explore the effects of a GABAA-α5 NAM (basmisanil) on intellectual disability associated with Down syndrome.

A randomized, double-blind, placebo-controlled phase II trial to explore the effects of a GABAA-α5 NAM (basmisanil) on intellectual disability associated with Down syndrome.

Journal: Journal of neurodevelopmental disorders

Published: August 10, 2021

Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls.

Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls.

Journal: The New England journal of medicine

Published: July 28, 2021

Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial.

Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial.

Journal: The Lancet. Neurology

Published: May 10, 2021

Safety of AADC Gene Therapy for Moderately Advanced Parkinson Disease: Three-Year Outcomes From the PD-1101 Trial.

Safety of AADC Gene Therapy for Moderately Advanced Parkinson Disease: Three-Year Outcomes From the PD-1101 Trial.

Journal: Neurology

Published: March 05, 2021

Risdiplam in Type 1 Spinal Muscular Atrophy.

Risdiplam in Type 1 Spinal Muscular Atrophy.

Journal: The New England journal of medicine

Published: February 24, 2021

Haematological effects of oral administration of bitopertin, a glycine transport inhibitor, in patients with non-transfusion-dependent β-thalassaemia.

Haematological effects of oral administration of bitopertin, a glycine transport inhibitor, in patients with non-transfusion-dependent β-thalassaemia.

Journal: British journal of haematology

Published: January 29, 2021

Correction to: Exploring Social Biomarkers in High‑Functioning Adults with Autism and Asperger's Versus Healthy Controls: A Cross‑Sectional Analysis.

Correction to: Exploring Social Biomarkers in High‑Functioning Adults with Autism and Asperger's Versus Healthy Controls: A Cross‑Sectional Analysis.

Journal: Journal of autism and developmental disorders

Published: May 06, 2020

Exploring Social Biomarkers in High-Functioning Adults with Autism and Asperger's Versus Healthy Controls: A Cross-Sectional Analysis.

Exploring Social Biomarkers in High-Functioning Adults with Autism and Asperger's Versus Healthy Controls: A Cross-Sectional Analysis.

Journal: Journal of autism and developmental disorders

Published: April 13, 2020

Severity Assessment in CDKL5 Deficiency Disorder.

Severity Assessment in CDKL5 Deficiency Disorder.

Journal: Pediatric neurology

Published: December 30, 2018

Showing 1-12 of 44

Last Updated: 09/26/2026