Genetic susceptibility to thrombotic microangiopathy in a 10-month-old patient with SMA type 1: a caveat for gene therapy eligibility.

doi:10.1016/j.nmd.2026.107376

Article

Genetic susceptibility to thrombotic microangiopathy in a 10-month-old patient with SMA type 1: a caveat for gene therapy eligibility.

Neuromuscular disorders : NMD - 1 Sept 2026

D'Alessandro Rossella, Somà Alessandra, Vacchetti Martina, Salvalaggio Anna, Guarnone Eleonora, Cavallina Ilaria, Rolle Enrica, Rossi Francesca, Montin Davide, Saglio Francesco, Peruzzi Licia, Barberis Marco, Sbaiz Luca, Deaglio Silvia, Fagioli Franca, Mongini Tiziana Enrica, Ricci Federica Silvia

Abstract excerpt

Gene therapy with onasemnogene abeparvovec (OA) has dramatically improved the prognosis of spinal muscular atrophy (SMA) since its approval. Thrombotic microangiopathies (TMA), including atypical complement-mediated hemolytic uremic syndrome (aHUS), represent rare but potentially life-threatening complications associated with OA. Complement activation and immune response against the viral vector capsid are...

Read the complete abstract on PubMed

Topics

Humans
Female
Infant
Thrombotic Microangiopathies
Genetic Predisposition to Disease

Genetic susceptibility to thrombotic microangiopathy in a 10-month-old patient with SMA type 1: a caveat for gene therapy eligibility.

Abstract excerpt

Topics

Related research