Rare Disease

November 8th 2024

Perspectives of patients with spinal muscular atrophy (SMA) and their caregivers should be weighed before making decisions around SMA-enhancing treatments.

October 28th 2024

Oral function should be measured routinely to determine individual bulbar involvement stages in spinal muscular atrophy (SMA).

October 17th 2024

Investigators say thrombospondin-4 levels were reduced in patients with symptoms of spinal muscular atrophy (SMA), but levels of the protein increased after therapy.

October 17th 2024

Patients aged 2-21 receiving apitegromab showed clinically meaningful motor function improvements, with a favorable safety profile consistent with long-term data, as Scholar Rock prepares for US and EU regulatory submissions in Q1 2025.