Vinay Penematsa

PTC Therapeutics (United States) — United States

Biochemistry, Genetics and Molecular Biology · Life Sciences

2h-index66citations13works0.02yr avg
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Research Topics

Muscle Physiology and Disorders(8), Cardiomyopathy and Myosin Studies(4), Metabolism and Genetic Disorders(3), Amino Acid Enzymes and Metabolism(2), Mitochondrial Function and Pathology(1)

Publications13 total

Pharmacodynamics, Efficacy, and Safety of Intraputaminal Eladocagene Exuparvovec Administered to Pediatric Patients With <scp>Aromatic L‐Amino Acid Decarboxylase</scp> Deficiency Using an <scp>MR</scp> ‐Compatible Cannula: 48 Weeks of Follow‐Up
Journal of Inherited Metabolic Disease·2026Open Access
Confirmatory long-term efficacy and safety results of ataluren in patients with nmDMD from Study 041, an international, randomized, double-blind, placebo-controlled, Phase III trial
Journal of Comparative Effectiveness Research·2025Open Access
O14: Administration of eladocagene exuparvovec using the SmartFlow magnetic resonance-compatible ventricular cannula in pediatric patients with aromatic ʟ-amino acid decarboxylase deficiency
Genetics in Medicine Open·2025Open Access
Ataluren Delays Clinically Meaningful Milestones of Decline in 6WMD in Patients with nmDMD from Study 041, a Phase 3, Placebo-controlled Trial (S21.001)
Neurology·2024
Ataluren Slows the Decline of Muscle Function in Patents with nmDMD: A Meta-analysis of Three Randomized, Double-blind, Placebo-controlled Trials (P3-11.002)
Neurology·2024
Pharmacodynamics of Eladocagene Exuparvovec and Safety of the SmartFlow Magnetic Resonance-compatible Ventricular Cannula for Administering Eladocagene Exuparvovec in Pediatric Participants (P10-3.005)
Neurology·2024
Supplementary tables: Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients
Figshare·2024Open Access
Ataluren Preserves Muscle Function in nmDMD Patients: A Pooled Analysis of Results from Three Randomized, Double-Blind, Placebo-Controlled Trials
Neuropediatrics·2023
Safety and Efficacy of Ataluren in nmDMD Patients from Study 041, a Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial (PL5.001)
Neurology·2023· 6 cited
Ataluren Preserves Upper Limb Function in nmDMD Patients from Study 041, a Phase 3 Placebo-Controlled Trial, and the STRIDE Registry (S34.008)
Neurology·2023· 1 cited
Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients
Journal of Comparative Effectiveness Research·2021· 58 citedOpen Access
Ataluren Delays Loss of Ambulation and Decline in Pulmonary Function in Patients with Nonsense Mutation Duchenne Muscular Dystrophy (2553)
Neurology·2021· 1 cited
DMD – THERAPY
Neuromuscular Disorders·2020

Frequent Co-authors

Christian Werner(9), Craig M. McDonald(7), Panayiota Trifillis(7), Connie Chou(6), Francesco Muntoni(5), Heather Gordish‐Dressman(5), M. Tulinius(5), Jeffrey Statland(5), Paula Williams(5), Joel Jiang(4), Francesco Bibbiani(4), Elizabeth Goodwin(4), Lauren P. Morgenroth(4), Peter Karachunski(4), Michelle Lorentzos(4), Kazuhiro Haginoya(4), Allan Kristensen(3), Richard Able(3), Shiwen Wu(3), Sheffali Gulati(3)