Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates

Kathrin Meyer, Laura Ferraiuolo, Leah Schmelzer, +11

Molecular Therapy

Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial

Eugenio Mercuri, Francesco Muntoni, Giovanni Baranello, +52

The Lancet Neurology

Gene TherapiesClinical TrialsNeurologyMedicinePhase 3

A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy

Jerry R. Mendell, Zarife Sahenk, Vinod Malik, +17

Molecular Therapy

Nusinersen safety and effects on motor function in adult spinal muscular atrophy type 2 and 3

Lorenzo Maggi, Luca Bello, Silvia Bonanno, +38

Journal of Neurology Neurosurgery & Psychiatry

Genotype and phenotype characterization in a large dystrophinopathic cohort with extended follow-up

Francesca Magri, Alessandra Govoni, Maria Grazia D’Angelo, +18

Journal of Neurology

Phenotype CharacterizationUrologyGenetic EpidemiologyDegenerative DiseaseLarge Dystrophinopathic Cohort +3

The italian limb girdle muscular dystrophy registry: Relative frequency, clinical features, and differential diagnosis

Francesca Magri, Vincenzo Nigro, C. Angelini, +30

Muscle & Nerve

Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial

Enrico Bertini, Éric Dessaud, Eugenio Mercuri, +36

The Lancet Neurology

Placebo-controlled Phase 2Chronic Musculoskeletal ConditionType 2Clinical TrialsSpinal Muscular Atrophy +4

Discontinuation of Primary Prophylaxis for<i>Pneumocystis carinii</i>Pneumonia and Toxoplasmic Encephalitis in Human Immunodeficiency Virus Type I–Infected Patients: The Changes in Opportunistic Prophylaxis Study

Cristina Mussini, Patrizio Pezzotti, Alessandra Govoni, +14

The Journal of Infectious Diseases

Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial

Riccardo Masson, Maria Mazurkiewicz-Bełdzińska, Kristy Rose, +166

The Lancet Neurology

Type 1Firefish Part 2RehabilitationNeurologySpinal Muscular Atrophy +4

Cidofovir added to HAART improves virological and clinical outcome in AIDS-associated progressive multifocal leukoencephalopathy

Andrea De Luca, Maria Letizia Giancola, Adriana Ammassari, +8

AIDS