Publications by authors named "Perry B Shieh"

Claim this Profile
T
Trach and treat: Safety and motor outcomes following onasemnogene abeparvovec in patients with spinal muscular atrophy and tracheostomies in the RESTORE registry.
Yasemin Erbaş, Laurent Servais, Perry B Shieh, Natalie L Goedeker, Megan A Waldrop

J Neuromuscul Dis· November 2025

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis
C
Cardiac safety of fordadistrogene movaparvovec gene therapy in Duchenne muscular dystrophy: Initial observations from a phase 1b trial.
Sarah P Sherlock, Daniel I Levy, Avery McIntosh, Perry B Shieh, Edward C Smith

Mol Ther· September 2025

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis
C
Complement activation in a phase Ib study of fordadistrogene movaparvovec for Duchenne muscular dystrophy.
Barry J Byrne, Russell J Butterfield, Perry B Shieh, Edward C Smith, Christoph Licht

Mol Ther· September 2025

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis
G
Gene therapy for children with X-linked myotubular myopathy: a plain language summary of publication for the ASPIRO study.
Perry B Shieh, Wendy Hughes, Marie Wood, Alan H Beggs, Michael W Lawlor

Ther Adv Rare Dis· September 2025

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis
C
Consensus recommendations and considerations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophy.
Jodi M Wolff, Nora Capocci, Evrim Atas, Diana X Bharucha-Goebel, John F Brandsema, Perry B Shieh

Neuromuscul Disord· September 2025

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis
A
AAV mini-dystrophin gene therapy for Duchenne muscular dystrophy: a phase 1b trial.
Russell J Butterfield, Perry B Shieh, Huihua Li, Michael Binks, Tara G McDonnell

Nat Med· August 2025

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis
S
Switching to subcutaneous zilucoplan from intravenous complement component 5 inhibitors in generalised myasthenia gravis: a phase IIIb, open-label study.
Miriam Freimer, Urvi Desai, Raghav Govindarajan, Min K Kang, Shaida Khan, Perry B Shieh

Ther Adv Neurol Disord· July 2025

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis
S
Strength and functional correlates of reachable workspace in facioscapulohumeral muscular dystrophy.
Leo H Wang, Maya N Hatch, Michael P McDermott, William B Martens, Katy Eichinger, Perry B Shieh

Neuromuscul Disord· March 2025

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis
E
Effect of immunomodulatory agents on the response to COVID-19 vaccination among patients with neuromuscular diseases: A single center experience.
Jaylin Hsu, Perry B Shieh

Medicine (Baltimore)· February 2025

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis
C
Current insights in ultra-rare adenylosuccinate synthetase 1 myopathy - meeting report on the First Clinical and Scientific Conference. 3 June 2024, National Centre for Advancing Translational Science, Rockville, Maryland, the United States of America.
Emma Rybalka, Hyung Jun Park, Atchayaram Nalini, Dipti Baskar, Kiran Polavarapu, Perry B Shieh

Orphanet J Rare Dis· November 2024

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis
H
Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy Therapies.
Ryan Fischer, Pat Furlong, Annie Kennedy, Kelly Maynard, Marissa Penrod, Perry B Shieh

J Multidiscip Healthc· August 2024

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis
S
Safety and efficacy of losmapimod in facioscapulohumeral muscular dystrophy (ReDUX4): a randomised, double-blind, placebo-controlled phase 2b trial.
Rabi Tawil, Kathryn R Wagner, Johanna I Hamel, Doris G Leung, Jeffrey M Statland, Perry B Shieh

Lancet Neurol· May 2024

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis
R
Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry.
Laurent Servais, John W Day, Darryl C De Vivo, Janbernd Kirschner, Eugenio Mercuri, Perry B Shieh

J Neuromuscul Dis· March 2024

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis
E
Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial.
Utkarsh J Dang, Jesse M Damsker, Michela Guglieri, Paula R Clemens, Seth J Perlman, Perry B Shieh

Neurology· March 2024

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis
R
Recurrent de novo variant causes childhood-onset amyotrophic lateral sclerosis (ALS) by excess sphingolipid synthesis.
Safoora B Syeda, Museer A Lone, Payam Mohassel, Sandra Donkervoort, Pinki Munot, Perry B Shieh

J Neurol Neurosurg Psychiatry· January 2024

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis
E
Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial.
Michael W Lawlor, Benedikt Schoser, Marta Margeta, Caroline A Sewry, Karra A Jones, Perry B Shieh

EBioMedicine· January 2024

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis
S
Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial.
Perry B Shieh, Nancy L Kuntz, James J Dowling, Wolfgang Müller-Felber, Carsten G Bönnemann

Lancet Neurol· December 2023

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis
E
Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy.
Jerry R Mendell, Perry B Shieh, Craig M McDonald, Zarife Sahenk, Kelly J Lehman

Front Cell Dev Biol· July 2023

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis
I
Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG).
Richard S Finkel, Basil T Darras, Jerry R Mendell, John W Day, Nancy L Kuntz, Perry B Shieh

J Neuromuscul Dis· May 2023

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis
D
Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status.
Craig M McDonald, Jessica R Marden, Perry B Shieh, Brenda L Wong, Henry Lane

J Comp Eff Res· April 2023

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis
E
Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
Michela Guglieri, Paula R Clemens, Seth J Perlman, Edward C Smith, Iain Horrocks, Perry B Shieh

JAMA Neurol· October 2022

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis
E
Experience and Perspectives in the US on the Evolving Treatment Landscape in Spinal Muscular Atrophy.
Leigh Ramos-Platt, Lauren Elman, Perry B Shieh

Int J Gen Med· September 2022

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis
Q
Quantitative Muscle Analysis in FSHD Using Whole-Body Fat-Referenced MRI: Composite Scores for Longitudinal and Cross-sectional Analysis.
Michelle L Mellion, Per Widholm, Markus Karlsson, André Ahlgren, Rabi Tawil, Perry B Shieh

Neurology· August 2022

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis
O
Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial.
Kevin A Strauss, Michelle A Farrar, Francesco Muntoni, Kayoko Saito, Jerry R Mendell, Perry B Shieh

Nat Med· July 2022

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis
O
Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial.
Kevin A Strauss, Michelle A Farrar, Francesco Muntoni, Kayoko Saito, Jerry R Mendell, Perry B Shieh

Nat Med· July 2022

Social Media Activity not collected for this article yet.

Sign Up to Request Social Media Analysis