Last Updated: 09/26/2026
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Found 36 publications
Author Correction: Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial.
Journal: Nature medicine
Published: January 28, 2026
Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial.
Journal: Nature medicine
Published: July 23, 2025
Intrathecal onasemnogene abeparvovec in treatment-naive patients with spinal muscular atrophy: a phase 3, randomized controlled trial.
Journal: Nature medicine
Published: July 23, 2025
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
The STARS Phase 2 Study: A Randomized Controlled Trial of Gaboxadol in Angelman Syndrome.
Journal: Neurology
Published: March 03, 2020
Clinical Development of Targeted Fragile X Syndrome Treatments: An Industry Perspective.
Journal: Brain sciences
Published: November 14, 2018
Adaptive behavior in adolescents and adults with Down syndrome: Results from a 6-month longitudinal study.
Journal: American journal of medical genetics. Part A
Published: January 16, 2018
A Retrospective, Longitudinal, Claims-Based Comparison of Concomitant Diagnoses Between Individuals with and Without Down Syndrome.
Journal: Journal of managed care & specialty pharmacy
Published: June 27, 2017
Pharmacological interventions to improve cognition and adaptive functioning in Down syndrome: Strides to date.
Journal: American journal of medical genetics. Part A
Published: December 23, 2016
Arbaclofen in fragile X syndrome: results of phase 3 trials.
Journal: Journal of neurodevelopmental disorders
Published: May 10, 2016
Parental Perspectives on Pharmacological Clinical Trials: a Qualitative Study in Down Syndrome and Fragile X Syndrome.
Journal: Journal of genetic counseling
Published: February 22, 2016
Last Updated: 09/26/2026