August 15th 2025
Children with spinal muscular atrophy (SMA) showed motor function improvements after switching to onasemnogene abeparvovec following prior treatment with nusinersen or risdiplam in a real-world study.
February 28th 2025
Raising Awareness of Rare Diseases and the Need for More Research
Survey Finds Varied Views on Value of Multiple Sclerosis Therapies
Experimental Treatment Induces Remission in Multiple Sclerosis
Rare Disease Information Websites Vary in Quality, Study Finds