Klopstock Thomas

🇣🇤 German Center for Neurodegenerative Diseases

2 articles in GJC

Five-Year Outcomes of Lenadogene Nolparvovec Gene Therapy in Leber Hereditary Optic Neuropathy.

Yu-Wai-Man Patrick, Newman Nancy J, Biousse Valérie, Carelli Valerio, Moster Mark L, Vignal-Clermont Catherine et al.

JAMA OphthalmolFeb 202511 citationsClinical Trial

Gene therapy for LHON (MT-ND4 variant) showed sustained bilateral visual improvement and good safety up to five years, offering promising long-term benefit for patients.

Full abstract PubMed

Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset.

Newman Nancy J, Yu-Wai-Man Patrick, Carelli Valerio, Moster Mark L, Biousse Valerie, Vignal-Clermont Catherine et al.

OphthalmologyJan 2021126 citationsRandomized Controlled Trial

This gene therapy study for early LHON found no significant visual acuity difference between treated and sham eyes, suggesting limited direct benefit from the injection itself.

Full abstract PubMed

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Klopstock Thomas

Five-Year Outcomes of Lenadogene Nolparvovec Gene Therapy in Leber Hereditary Optic Neuropathy.

Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset.