News

Cure SMA’s Early-Stage Funding Leads to Big Pharma Investments in Promising Therapies

Cure SMA, an organization founded in 1984 to fund and invest in research for spinal muscular atrophy (SMA) treatments, also focuses on the day-to-day realities of patients and families, reaching about 4,000 SMA families every year through family support services with its 110,000-plus members and supporters. Among its other milestones, Cure SMA has invested more…

Blocking a Spinal Muscular Atrophy Enzyme, JNK3, Seen to Ease Disease in Mice Regardless of Underlying Mutation

Texas Tech University researchers have identified a possible therapeutic target, the enzyme JNK3, for spinal muscular atrophy (SMA) that is independent of the causing genetic mutation. Their research, titled “Genetic Inhibition of JNK3 Ameliorates Spinal Muscular Atrophy,” was published in Human Molecular Genetics. SMA is a genetic neurodegenerative…