Melissa SantaMaria

Risdiplam treatment has not led to retinal toxicity in patients with spinal muscular atrophy

E. Mercuri, R. Sergott, S. Nave, 2020, Annals of clinical and translational neurology.

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

J. Sahel, M. Neuenhahn, N. Newman, 2021, Ophthalmology.

Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients with Leber Hereditary Optic Neuropathy Carrying the m.11778G>A MT-ND4 Mutation

Shriji N. Patel, J. Sahel, M. Neuenhahn, 2022, Ophthalmology and Therapy.