Evrysdi (risdiplam) effectively improved motor function in adults with spinal muscular atrophy (SMA) type 2 or type 3,…
Articles by Andrea Lobo
Young children with severe spinal muscular atrophy (SMA) have substantial loss of motor neurons in the thoracic region of…
The muscle fibers of children with spinal muscular atrophy (SMA) type 1 are substantially different from those of age-matched…
NIDO-361, Nido Biosciences’ experimental oral treatment for spinal and bulbar muscular atrophy (SBMA) — a disease type marked…
The Ministry of Health, Labour, and Welfare (MHLW) in Japan has extended the approval of Evrysdi (risdiplam) for infants…
Substantial differences in newborn screening (NBS) practices for spinal muscular atrophy (SMA) across the U.S. could impact referral patterns…
Patients with spinal muscular atrophy (SMA) in China and their caregivers have low levels of health information literacy, or…
An early spinal muscular atrophy (SMA) diagnosis with the help of a newborn screening program, and treatment with disease-modifying…
An 8-month-old girl with a clinical and genetic diagnosis of spinal muscular atrophy (SMA) type 1 saw her…
A young girl whose muscle weakness and respiratory distress in infancy were treated only as such was diagnosed as a…