Author Archives: Michela Luciano, PhD

Prenatal therapy extends survival in SMA mice, study finds

A one-time treatment designed to correct a single letter, or base, in the SMN2 gene increased levels of the survival motor neuron (SMN) protein — which is lacking in spinal muscular atrophy (SMA) — and prolonged survival in…

Scientist awarded for breakthrough RNA research that led to Spinraza

A New York-based scientist has been awarded for his pioneering work that led to the development of Spinraza (nusinersen), the first RNA-based therapy to target the underlying cause of spinal muscular atrophy (SMA). Adrian R. Krainer,…

Blood creatinine-to-cystatin-c ratio may track SMA treatment response

Blood creatinine-to-cystatin-c ratio, an emerging indicator of muscle mass and strength, may be a promising biomarker of therapeutic response in adults with spinal muscular atrophy (SMA), a study in China shows. Increases in the ratio were significantly associated…

St. Jude pediatric neurologist included on Time100 health list

Richard Finkel, MD, a pediatric neurologist at St. Jude Children’s Research Hospital, was included on a list of 100 individuals having a positive influence on global health in 2025, in recognition of his work treating childhood neuromuscular disorders…