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Combinations of ASOs for the Modulation of SMN Expression in SMA Patients

Unmet NeedSpinal muscular atrophy (SMA) is a devastating neuromuscular disease that predominantly affects children and is the most common cause of hereditary infant mortality. The disease is caused by low levels of survival motor neuron (SMN) protein, due to recessive mutations in the SMN1 gene. Importantly, the copy number variation of SMN2, a homologous...

Published: 3/13/2026 | Inventor(s): Charlotte Sumner, Constantin d'Ydewalle, Frank Rigo, C. Bennett

Keywords(s): CNS and Neurological Disorders, Combination, Disease Indication, Drug Delivery Vehicle, Muscle Atrophy, Muscular Disorders, Neurodegenerative Diseases, Peptides/Prodrugs, Single, Targeted Therapy, Therapeutic Matter, Therapeutics, Therapy Type

Category(s): Clinical and Disease Specializations > Neurology > Motor Neuron Diseases, Technology Classifications > Therapeutic Modalities > Therapeutic Delivery Platforms, Technology Classifications > Therapeutic Modalities > Proteins